Nationwide Children's News Releases
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May 28, 2025
Nationwide Children’s Hospital Delivers its First Infusion of CASGEVY™ for Patient with Sickle Cell Disease
Nationwide Children’s Hospital delivered its first commercial infusion of CASGEVY™ to 17-year-old Carlos A. from Dayton, Ohio. Today, Carlos is thriving at home, pain-free.
Feb 03, 2025
Nationwide Children’s Hospital Researcher Receives Grant from the Rosenau Family Research Foundation
The Rosenau Family Research Foundation has awarded a three-year grant of $375,000 total in funding to Allison Bradbury, MS, PhD, along with collaborators at Cincinnati Children’s Hospital Medical Center and Auburn University, for studying in-utero gene therapy as a potential treatment for Krabbe disease and other lysosomal storage diseases.
Nov 07, 2024
Nationwide Children’s Hospital Receives a Wellstone Muscular Dystrophy Specialized Research Center Award from the NIH
Researchers in the Jerry R. Mendell, MD Center for Gene Therapy at Nationwide Children's Hospital have received a coveted P50 grant from the National Institutes of Health to establish a Wellstone Muscular Dystrophy Specialized Research Center (MDSRC).
Sep 10, 2024
Nationwide Children’s Hospital Renames Center for Gene Therapy, Honoring Jerry Mendell, MD
Nationwide Children’s Hospital announced today the renaming and dedication of the Jerry R. Mendell, MD Center for Gene Therapy at the Abigail Wexner Research Institute, in honor of Jerry Mendell, MD, a pioneer in the fields of neurology and gene therapy and a catalyst for advancement in the treatment of multiple neuromuscular disorders.
Jun 14, 2024
Nationwide Children’s Hospital Receives $25 Million Gift to Support Institute for Mental and Behavioral Health Research
Nationwide Children’s Hospital announced today that longtime donors Joe and Linda Chlapaty have donated $25 million to support the recently launched Institute for Mental and Behavioral Health Research. The gift will fund innovative research, endowed faculty to recruit nationally renowned researchers, and construction for new facilities.
Jan 18, 2024
Jerry Mendell, MD, Announced as King Faisal Prize Laureate in Medicine
Jerry Mendell, MD, senior advisor in the Center for Gene Therapy in the Abigail Wexner Research Institute at Nationwide Children’s Hospital, is this year’s recipient of the King Faisal Prize for Medicine for his contributions toward the screening, diagnosis and treatment of neuromuscular diseases, including spinal muscular atrophy (SMA), Duchenne muscular dystrophy (DMD) and limb girdle muscular dystrophy.
Dec 11, 2023
Nationwide Children’s Hospital Becomes Authorized Treatment Center for Sickle Cell Gene Therapy
Nationwide Children’s Hospital has been designated an authorized treatment center for CASGEVY™ (exagamglogene autotemcel (exa-cel)), a gene therapy for the treatment of sickle cell disease.
Aug 08, 2023
From Bench to Bedside: Patient with Duchenne Muscular Dystrophy Receives First Dose of Gene Therapy Developed at Nationwide Children’s Hospital
In a landmark moment for the Abigail Wexner Research Institute at Nationwide Children’s, a 5-year-old from Bellefontaine, Ohio, received the first dose of a recently approved gene therapy for Duchenne muscular dystrophy at Nationwide Children’s Hospital, where the therapy was invented and initially tested.
Jun 22, 2023
FDA Approves First Gene Therapy Treatment for Duchenne Muscular Dystrophy Developed at the Abigail Wexner Research Institute at Nationwide Children’s Hospital
Today, the Abigail Wexner Research Institute at Nationwide Children’s Hospital praised the Food and Drug Administration (FDA) for its accelerated approval of SRP-9001/ELEVIDYS for Duchenne muscular dystrophy (DMD) following decades of research in its Center for Gene Therapy to help patients with neuromuscular diseases.
May 19, 2023
Nationwide Children’s Hospital Becomes Certified Site for Gene Therapy for Transfusion-Dependent Beta Thalassemia
Nationwide Children’s Hospital has been designated a qualified treatment center for Zynteglo® (betibeglogene autotemcel), a a gene therapy approved for the treatment of people with beta thalassemia who require regular red blood cell transfusions.
Jan 20, 2023
New Insights into the Role of the NOTCH1 Gene in Congenital Heart Defects
A new study published in Circulation Research provides novel insights into how mutations in the NOTCH1 gene may lead to abnormal differentiation and proliferation of cardiomyocytes and contribute to ventricular hypoplasia and other structural anomalies found in congenital heart defects in humans.
Aug 18, 2022
New Disorder of Copper Metabolism Identified, Caused by Variants in CTR1 Gene
Copper is essential for many cellular functions, including cellular respiration, antioxidant defense, neurotransmitter biosynthesis and neuropeptide amidation, among others. Until recently, only two inborn errors of copper metabolism were well established. Both are rare. Wilson's disease and Menkes disease result from mutations in the copper-transporting P-type ATPases; ATP7B and ATP7A, respectively.
Mar 09, 2022
Early Phase Gene Therapy Study for SMARD1/CMT2S Disease Now Enrolling
Nationwide Children's Hospital has launched a new Phase I/IIa gene replacement clinical study to evaluate the safety of a gene therapy for IGHMBP2-related diseases, specifically spinal muscular atrophy with respiratory distress type 1 (SMARD1) and Charcot Marie Tooth disease type 2S (CMT2S). SMARD1 and CMT2S are rare inherited conditions that are both caused by mutations in the IGHMBP2 gene, of which more than 60 have been found to date. SMARD1 is the most severe form of these diseases and causes muscle weakness and respiratory failure in infancy.
Feb 25, 2022
Dennis Durbin, MD, MSCE, Named President of the Abigail Wexner Research Institute at Nationwide Children’s Hospital
Beginning March 1, 2022, Dennis Durbin, MD, MSCE, will become the third president of the Abigail Wexner Research Institute at Nationwide Children's Hospital. Dr. Durbin joined Nationwide Children's in 2018 as its first chief scientific officer.
Jan 26, 2022
Nationwide Children’s Hospital Researcher Lauren Bakaletz, PhD, Honored by AAAS as Lifetime AAAS Fellow
The American Association for the Advancement of Science (AAAS), the world’s largest general scientific society and publisher of the Science family of journals, has elected Lauren Bakaletz, PhD, director of the Center for Microbial Pathogenesis in the Abigail Wexner Research Institute (AWRI) at Nationwide Children’s Hospital to the newest class of AAAS Fellows, among the most distinct honors within the scientific community.
Dec 13, 2021
Human MicroRNA Inhibits Expression of Pathogenic Gene Underlying Facioscapulohumeral Muscular Dystrophy
Facioscapulohumeral muscular dystrophy (FSHD) is caused by aberrant expression of the DUX4 gene in skeletal muscles. Researchers at Nationwide Children’s Hospital have recently demonstrated that an endogenous human microRNA, miR-675, inhibits DUX4 expression and protects muscles from DUX4-mediated cell death in a mouse model when administered via gene therapy. They also showed that the small molecule-based treatments that upregulate miR-675 inhibited DUX4 mRNA and DUX4-associated biomarkers in myotubes derived from patients with FSHD.
Mar 18, 2021
Nationwide Children’s Hospital Affiliate Andelyn Biosciences Receives Significant Investment and Enters Into Strategic Partnership with Pall Corporation and Cytiva
(COLUMBUS, Ohio) – Andelyn Biosciences, Inc, a contract development and manufacturing organization (CDMO) and an affiliate company of Nationwide Children’s Hospital, has received a significant, minority investment from Pall Corporation.
Nov 23, 2020
Andelyn Biosciences Breaks Ground for New Commercial-Scale Gene Therapy Manufacturing Facility
Andelyn Biosciences, an affiliate company of Nationwide Children’s Hospital that manufactures gene therapy products for the biotechnology and pharmaceutical industries, has broken ground on its new site located at 1250 Arthur E. Adams Dr. in The Ohio State University’s Innovation District. Opening in 2022, it will be central Ohio’s first commercial-scale Good Manufacturing Practices (cGMP) production facility devoted to gene therapies.
Oct 19, 2020
Jerry R. Mendell, MD, Elected to the Prestigious National Academy of Medicine
(COLUMBUS, Ohio) – Today, Jerry R. Mendell, MD, principal investigator in the Center for Gene Therapy at the Abigail Wexner Research Institute at Nationwide Children’s Hospital, was elected to the National Academy of Medicine (NAM), the second faculty member from Nationwide
May 01, 2020
Nationwide Children’s Hospital Names CEO of Andelyn Biosciences
Nationwide Children's Hospital is pleased to announce Mayo Pujols as CEO of Andelyn Biosciences, an affiliate company that will manufacture gene therapy products for the biotechnology and pharmaceutical industries.
Jan 13, 2020
Nationwide Children’s Hospital Announces Plans for Andelyn Biosciences
Central Ohio’s first commercial-scale gene therapy Good Manufacturing Practices (cGMP) clinical manufacturing facility slated for 2023.
May 29, 2019
Nationwide Children’s Hospital Named a CMT Center of Excellence
The Charcot-Marie-Tooth Association has named Nationwide Children’s Hospital a CMT Center of Excellence, in recognition of the comprehensive care and expertise provided to children with the most common form of Charcot-Marie-Tooth disease, CMT1A.
May 24, 2019
FDA Approves First Gene Therapy Treatment for SMA Developed at the Abigail Wexner Research Institute at Nationwide Children’s Hospital
Today, the Abigail Wexner Research Institute at Nationwide Children’s Hospital praised the Food and Drug Administration (FDA) for its approval of Zolgensma® for spinal muscular atrophy (SMA) following decades of research in its Center for Gene Therapy to help patients with neuromuscular diseases.
May 21, 2019
Nationwide Children’s Hospital Dedicates Abigail Wexner Research Institute at Nationwide Children’s Hospital
Nationwide Children’s Hospital dedicated the hospital’s Research Institute as the Abigail Wexner Research Institute at Nationwide Children’s Hospital. This naming recognizes Mrs. Wexner, whose ongoing, passionate advocacy has ushered in a period of unprecedented transformation at the hospital.
May 20, 2019
MEDIA ADVISORY: Dedication of the Abigail Wexner Research Institute at Nationwide Children’s Hospital
WHAT: The dedication of the Abigail Wexner Research Institute at Nationwide Children’s Hospital and announcement of the Allen Distinguished Scholar in Pediatric Research WHEN: Tuesday, May 21, 2019 10:30 a.m. – Media Tour of the Abigail Wexner Research Institute 11:30 a.m.
Apr 01, 2019
Anne M. Connolly, MD, Named Division Chief of Neurology at Nationwide Children’s Hospital
Anne M. Connolly, MD, has joined Nationwide Children’s Hospital as division chief of Neurology and a member of the Center for Gene Therapy.
Jan 25, 2019
Nationwide Children’s Hospital Announces Plans to Dedicate the Abigail Wexner Research Institute
Ms. Wexner’s partnership with her fellow board members, Chief Executive Officer Steve Allen, MD, and the Nationwide Children’s executive team made this evolution possible. In recognition of Dr. Allen’s role, Nationwide Children’s will also establish the Allen Distinguished Scholar in Pediatric Research.
Apr 24, 2018
Nationwide Children’s Researcher Wins Distinguished Clinical Research Achievement Award
The Clinical Research (CR) Forum, a non-profit membership association of top clinical research experts and thought leaders from the nation’s leading academic health centers, presented the Distinguished Clinical Research Achievement Award to two outstanding studies at the Top Ten Clinical Research Achievement Awards on April 18th at the National Press Club in Washington, D.C.
Mar 27, 2018
Nationwide Children's Hospital Study Named Among Top Ten Clinical Research Achievement Awards by the Clinical Research Forum
The first clinical trial to test the functional replacement of the mutated gene responsible for SMA1 will be recognized at the 2018 Top Ten Clinical Research Achievement Awards at the National Press Club in Washington, D.C., on April 18, 2018.
Nov 01, 2017
Phase 1 Study Shows Encouraging Data for Gene Replacement Therapy for Spinal Muscular Atrophy Type 1
A one-time intravenous infusion of the high dose of gene therapy extended the survival of patients with spinal muscular atrophy type 1 (SMA1) in a Phase 1 clinical trial, according to a study published today in the New England Journal of Medicine. The study was conducted by Researchers from Nationwide Children’s Hospital in collaboration with AveXis, Inc. and The Ohio State University College of Medicine.
Jan 10, 2017
Sarepta Therapeutics Enters Into License Agreement with Nationwide Childrens Hospital for Galgt2 Gene Therapy Program
Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a commercial stage developer of innovative RNA-targeted therapeutics, today announced it has entered a license agreement with Nationwide Children’s Hospital, for their Galgt2 gene therapy program developed by researcher Paul Martin, PhD.
Jan 10, 2017
Sarepta Therapeutics Enters Into Research Agreement and Option Agreement with Nationwide Childrens Hospital for Microdystrophin Gene Therapy Program
Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a commercial stage developer of innovative RNA-targeted therapeutics, today announced it has entered a research agreement with Nationwide Children’s Hospital on their microdystrophin gene therapy program.
Jan 03, 2017
Flanigan Named to Neuromuscular, Gene Therapy Leadership Roles
Beginning January 1, 2017, Kevin Flanigan, MD, will become the director of the Center for Gene Therapy and the Neuromuscular Disorders program at Nationwide Children’s Hospital. Dr.
Oct 12, 2016
Nationwide Childrens Hospital Neuromuscular Disease Researchers Receive $7.5 Million NIH Grant
The National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) of the National Institutes of Health announced four new Centers of Research Translation awards today.
Oct 04, 2016
Perky Jerky® to Support Nationwide Childrens Hospital Beginning in October
Nationwide Children’s Hospital and Perky Jerky® announced today that from October through December, customers at 16 participating Kroger stores will have an opportunity to donate to Nationwide Children’s by purchasing Perky Jerky®.
Sep 26, 2016
Oligodendrocytes Induce Motor Neuron Death in ALS
A first-of-its-kind oligodendrocyte in vitro model shows that human cells normally supportive of motor neuron function play an active role in amyotrophic lateral sclerosis pathogenesis – and this discovery may point the way toward therapeutic timing and targets.
Sep 20, 2016
Nationwide Childrens Hospital Leads Effort for Gene Therapy Treatment for Duchenne Muscular Dystrophy
Sarepta Therapeutics announced yesterday that the FDA has granted accelerated approval for eteplirsen for the treatment of Duchenne muscular dystrophy (DMD). Nationwide Children's Hospital was the sole original site for the clinical study that resulted in this approval, led by Dr.
Jul 20, 2016
AveXis Receives U.S. FDA Breakthrough Therapy Designation for AVXS-101 Gene Replacement Therapy for Spinal Muscular Atrophy Type 1
AveXis, Inc., (Nasdaq:AVXS) a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced the U.S.
Jul 01, 2016
Dean Lee, MD, PhD, to Lead Cell Therapy Programs at Nationwide Childrens Hospital and The Ohio State University Comprehensive Cancer Center
Dean Lee, MD, PhD, has been named the director of the Cellular Therapy and Cancer Immunotherapy Program for Nationwide Children’s Hospital’s Division of Hematology/Oncology/BMT and Center for Childhood Cancer and Blood Diseases. Dr.
Feb 29, 2016
Loss of MHCI in Motor Neurons Leads to ALS Astrocyte Toxicity
Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, is a devastating progressive neurodegenerative disease that results in the death of motor neurons, the nerve cells that control muscles.
Dec 01, 2015
Nationwide Children's Hospital Researcher, Mark Peeples, PhD, Elected as AAAS Fellow
Mark Peeples, PhD, principal investigator in the Center for Vaccines and Immunity at Nationwide Children’s Hospital, has been elected a Fellow in the American Association for the Advancement of Science (AAAS). Dr.
Jul 08, 2015
Dr. John Barnard Expands Leadership Role At Nationwide Children's Hospital And The Ohio State University
John A. Barnard, MD, President of The Research Institute at Nationwide Children’s Hospital, has been appointed both Chair of the Department of Pediatrics at The Ohio State University College of Medicine and Chief of Pediatrics at Nationwide Children’s Hospital.
May 07, 2015
Cure SMA Provides Additional Funding for Promising Spinal Muscular Atrophy Research at Nationwide Childrens Hospital
Cure SMA, the leading organization dedicated to the treatment and cure of spinal muscular atrophy (SMA), recently provided an additional $445,000 grant toward a gene therapy for SMA at Nationwide Children’s Hospital.
Apr 03, 2015
Cure Duchenne to Host a Webinar on Duplication Research for Duchenne Muscular Dystrophy with Dr. Kevin Flanigan from Nationwide Childrens Hospital on April 6
NEWPORT BEACH, Calif. -- CureDuchenne, a nonprofit that raises awareness and funds research to find a cure for Duchenne muscular dystrophy, will host an “Update on Duplication Mutation Research for Duchenne Muscular Dystrophy” webinar with Dr.
Aug 11, 2014
Discovery of New Form of Dystrophin Protein Could Lead to Therapy for Some DMD Patients
Scientists have discovered a new form of dystrophin, a protein critical to normal muscle function, and identified the genetic mechanism responsible for its production. Studies of the new protein isoform, published online Aug.
May 27, 2014
Nationwide Childrens Hospital receives $10 million gift from the Nationwide Insurance Foundation establishing the Nationwide Pediatric Innovation Fund
Nationwide Children’s Hospital today announced that it has received a $10 million gift from the Nationwide Insurance Foundation. The gift will establish the Nationwide Pediatric Innovation Fund at Nationwide Children’s Hospital, supporting pediatric research and innovative projects
Mar 26, 2014
Study Identifies Key Player in Motor Neuron Death in Lou Gehrigs Disease
Amyotrophic lateral sclerosis, also known as Lou Gehrig’s disease, is marked by a cascade of cellular and inflammatory events that weakens and kills vital motor neurons in the brain and spinal cord. The process is complex, involving cells that ordinarily protect the neurons from harm.
Mar 20, 2014
Parent Project Muscular Dystrophy Awards Nationwide Childrens Hospital; Names Hospital Certified Duchenne Care Center
Nationwide Children’s Hospital was named the first Certified Duchenne Care Center by Parent Project Muscular Dystrophy (PPMD), the leading advocacy organization working to end Duchenne muscular dystrophy (Duchenne).
Dec 03, 2013
Dr. Edwin Horwitz Appointed Director of Blood and Marrow Transplant at Nationwide Childrens Hospital
Edwin M. Horwitz, MD, PhD, recently was appointed director of Blood and Marrow Transplant (BMT) at Nationwide Children’s Hospital. Dr. Horwitz, board-certified in Pediatric Hematology/Oncology, comes to Nationwide Children’s from the Children’s Hospital of Philadelphia (CHOP).
Nov 25, 2013
Two Faculty in The Research Institute at Nationwide Childrens Hospital Named AAAS Fellows
Brian K. Kaspar, PhD, and Veronica J. Vieland, PhD, principal investigators in The Research Institute at Nationwide Children’s Hospital, have been named Fellows of the American Association for the Advancement of Science (AAAS).
Oct 30, 2013
Study: Researchers Identify Way to Increase Gene Therapy Success
Scientists in The Research Institute at Nationwide Children’s Hospital have found a way to overcome one of the biggest obstacles to using viruses to deliver therapeutic genes: how to keep the immune system from neutralizing the virus before it can deliver its genetic payload.
Oct 18, 2013
AveXis- BioLife Licenses Spinal Muscular Atrophy (SMA) Patent Portfolio from Nationwide Childrens Hospital and The Ohio State University
BioLife, a synthetic biology platform company soon to be renamed AveXis, has been granted exclusive rights to the spinal muscular atrophy gene therapy program developed at The Research Institute at Nationwide Children’s Hospital and The Ohio State University in Columbus, Ohio.
Oct 17, 2013
AveXis and BioLife Announce The Research Institute at Nationwide Childrens Hospital Received Fast Track Status for Spinal Muscular Atrophy Treatment
AveXis and BioLife, synthetic biology platform companies, today announced that The Research Institute at Nationwide Children’s Hospital received Fast Track designation from the U.S.
Oct 08, 2013
$25.4 Million Awarded to Center for Clinical and Translational Science to Continue Critical Bench to Bedside Translational Research
The National Institutes of Health has awarded a $25.4 million grant to the Center for Clinical and Translational Science (CCTS), a collaboration between The Ohio State University and Nationwide Children’s Hospital created to accelerate basic science discoveries into life-saving medical
Sep 09, 2013
Therapy Slows Onset and Progression of Lou Gehrigs Disease, Study Finds
Studies of a therapy designed to treat amyotrophic lateral sclerosis (ALS) suggest that the treatment dramatically slows onset and progression of the deadly disease, one of the most common neuromuscular disorders in the world.
Aug 09, 2013
Combined Therapy Could Repair and Prevent Damage in Duchenne Muscular Dystrophy
New research on two promising gene therapies suggests that combining them into one treatment not only repairs muscle damage caused by Duchenne muscular dystrophy, but also prevents future injury from the muscle-wasting disease.
Aug 08, 2013
New Drug Improves Walking Performance for Duchenne Muscular Dystrophy Patients
Results from a clinical trial of eteplirsen, a drug designed to treat Duchenne muscular dystrophy, suggest that the therapy allows participants to walk farther than people treated with placebo and dramatically increases production of a protein vital to muscle growth and health.
May 07, 2013
Family Foundations Fund Critical Study Necessary For Clinical Trials And Enter Into Agreement With Nationwide Childrens Hospital To Ensure Data Access For Lysosomal Storage Disease MPS III
Nearly 50 years to the day since Dr. Sylvester Sanfilippo first characterized the disease Mucopolysaccharidosis (MPS) III in a presentation to the American Pediatric Society, three parent-run foundations have awarded investigators at Nationwide Children’s Hospital in Columbus, OH with a
Apr 18, 2013
Dr. Brian Kaspar First Recipient of Grant Morrow, III, MD, Endowed Chair in Pediatric Research at Nationwide Childrens Hospital
During a recent ceremony, Brian Kaspar, PhD, was named the first recipient of the Grant Morrow, III, MD, Endowed Chair in Pediatric Research at Nationwide Children’s Hospital. This endowed chair is supported by Arthur E. Shepard and Dorothy D.
Mar 13, 2013
Families of SMA and Nationwide Childrens Hospital Announce Multi-Million Dollar Award from NINDS to Advance CNS-Directed Gene Therapy for Spinal Muscular Atrophy
Nationwide Children’s Hospital and Families of Spinal Muscular Atrophy (Elk Grove Village, IL) announce the award of a multi-million dollar cooperative agreement from the National Institute of Neurological Disorders and Stroke (NINDS) to advance a gene therapy development program for Spinal
Feb 18, 2013
2013 Technology Showcase Presented by The Research Institute at Nationwide Children's Hospital
The Office of Technology Commercialization in The Research Institute at Nationwide Children’s Hospital will present the “2013 Technology Showcase” Thursday, March 14, 2013 from 1 to 4 p.m.
Jan 31, 2013
Sophias Cure Foundation Donates $650,000 in 2012 to Nationwide Childrens Hospital
Brian Kaspar, PhD, principal investigator in the Center for Gene Therapy at The Research Institute at Nationwide Children’s Hospital, along with a team of Spinal Muscular Atrophy (SMA) researchers and clinicians, received $650,000 in grants in 2012 from Sophia’s Cure Foundation for SMA
Dec 07, 2012
Dr. John Barnard First Recipient of Ann I. Wolfe Endowed Chair in Research Leadership at Nationwide Children's Hospital
During the December 6 meeting of the Joint Boards of Directors at Nationwide Children’s Hospital, John Barnard, MD, President of The Research Institute at Nationwide Children’s was announced as the first recipient of the Ann I. Wolfe Endowed Chair in Research Leadership.
Oct 25, 2012
Mouse Model Could Help Identify Viral Vectors that May Cause Tumors
Investigators at Nationwide Children’s Hospital have identified a mouse model that could help evaluate the risk that viral vectors used in gene therapy might promote tumor formation as a side-effect. The study appears in Molecular Therapy.
Oct 05, 2012
Exon-Skipping Shows Promise in Duchenne Muscular Dystrophy
Results from a Phase IIb extension trial of the drug eteplirsen show an increased ability to walk in boys with Duchenne muscular dystrophy Eteplirsen, a drug that skips exon 51 of the dystrophin gene, may improve quality of life for patients with Duchenne muscular dystrophy and slow disease
Jul 23, 2012
RNA Interference May Provide the First Therapy for Facioscapulohumeral Muscular Dystrophy
Scientists may one day be able to treat the third most common muscular dystrophy, facioscapulohumeral muscular dystrophy (FSHD), by taking advantage of a system within living cells that helps control gene activation.
Jul 09, 2012
New Gene Transfer Strategy Shows Promise for Limb Girdle and Other Muscular Dystrophies
The challenge of treating patients with genetic disorders in which a single mutated gene is simply too large to be replaced using traditional gene therapy techniques may soon be a thing of the past.
Jun 21, 2012
Nationwide Childrens CEO to Co-Chair Parent Project Muscular Dystrophy Event
Nationwide Children’s Hospital CEO Steve Allen, MD, will serve as co-chair of the first Transforming Duchenne Care workshop to be held June 27 and 28, 2012, in Ft. Lauderdale, FL.
May 21, 2012
The 5th Annual Megans Miles Run/Walk to be Held May 27
The 5th Annual Megan’s Miles Run/Walk, benefitting Muscular Dystrophy research at Nationwide Children’s Hospital, will be held on Sunday, May 27 at noon at Central Crossing High School.
May 08, 2012
Dr. Timothy Cripe of Nationwide Childrens Hospital Appointed to a Committee of the Food and Drug Administration
Timothy Cripe, MD, PhD, chief of Hematology/Oncology/Bone Marrow Transplantation at Nationwide Children’s Hospital, was recently appointed a member of the Cellular, Tissue and Gene Therapy Advisory Committee which reports to the Food and Drug Administration (FDA).
Mar 19, 2012
Newborn Screening for Duchenne Muscular Dystrophy Shows Promise as an International Model
Investigators at Nationwide Children’s Hospital, working with the DNA Sequencing Core Facility at the University of Utah, have developed an approach to newborn screening (NBS) for the life-threatening genetic disorder, Duchenne muscular dystrophy (DMD) and potentially other muscular
Feb 29, 2012
Nationwide Childrens Hospital Neuromuscular Disorder Podcasts Now Available on iTunes
In 2010, the Center for Gene Therapy at Nationwide Children’s Hospital launched a monthly podcast entitled, “This Month in Muscular Dystrophy,” featuring internationally known scientists discussing the latest research in muscular dystrophy and other neuromuscular disorders.
Feb 22, 2012
Study: Muscle Regeneration May Provide Ideal Environment for Rhabdomyosarcoma
Inflammation, cell division and cell differentiation that occur during skeletal muscle regeneration may provide an ideal environment for the highly malignant tumor, rhabdomyosarcoma to arise.
Feb 15, 2012
Grant Provides Funding Toward Gene Therapy Clinical Trial to Replace the DMD Gene in All of the Leg Muscles of Duchenne Patients
Investigators in the Center for Gene Therapy in The Research Institute at Nationwide Children’s Hospital are working toward an approach to replace the defective gene in Duchene muscular dystrophy (DMD) by treating all of the muscles in the leg, thanks to funding from Jesse’s Journey.
Nov 02, 2011
Nationwide Children's Hospital and Ohio State Researchers Design a Viral Vector to Treat a Genetic Form of Blindness
Researchers at Ohio State University Medical Center and Nationwide Children’s Hospital have developed a viral vector designed to deliver a gene into the eyes of people born with an inherited, progressive form of blindness that affects mainly males.
Oct 03, 2011
Nationwide Childrens Hospital Researchers Receive Neurology Award for Contribution to Clinical Neuroscience
Jerry Mendell, MD, director of the Center for Gene Therapy in The Research Institute at Nationwide Children’s Hospital, and his fellow researchers, are the latest recipients of the Annals of Neurology prize for an outstanding contribution to clinical neuroscience. The study which helped Dr.
Aug 10, 2011
Human-Cell-Derived Model of ALS Provides a New Way to Study the Majority of Cases
For decades, scientists have studied a laboratory mouse model that develops signs of the paralyzing disease amyotrophic lateral sclerosis (ALS) as they age.
May 25, 2011
Nationwide Childrens Hospital Researchers Receive NIH Grant for the Move Toward Clinical Trials Targeting the Lysosomal Storage Disease MPSIIIB
Investigators at Nationwide Children’s have received a grant from the National Institutes of Health (NIH) to help move a therapy for MPS IIIB that has been shown effective in mice toward clinical trials in humans.
Jan 10, 2011
Patient-Derived Embryonic Stem Cells Help Deliver Good Genes in a Model of Inherited Blood Disorder
Researchers at Nationwide Children’s Hospital report a gene therapy strategy that improves the condition of a mouse model of an inherited blood disorder, Beta Thalassemia. The gene correction involves using unfertilized eggs from afflicted mice to produce a batch of embryonic stem cell lines.
Oct 06, 2010
Gene Therapy Reveals Unexpected Immunity to Dystrophin in Patients with Duchenne Muscular Dystrophy
An immune reaction to dystrophin, the muscle protein that is defective in patients with Duchenne muscular dystrophy, may pose a new challenge to strengthening muscles of patients with this disease, suggests a new study appearing in the October 7, 2010, issue of The New England Journal of Medicine.
Sep 08, 2010
Spinal Muscular Atrophy Research Team Receives Pepsi Refresh Funds from Sophias Cure Foundation
Brian Kaspar, PhD, principal investigator in the Center for Gene Therapy at The Research Institute at Nationwide Children’s Hospital, along with a team of Spinal Muscular Atrophy (SMA) researchers and clinicians, recently received a $250,000 grant for SMA research and clinic development from
Aug 26, 2010
$7 Million NIH Grant Designates Nationwide Childrens as a Wellstone Center to Advance Muscular Dystrophy Research
A $7 million grant from the National Institutes of Health (NIH) will help researchers at Nationwide Children’s Hospital translate new scientific findings and technological developments into novel treatments for the muscular dystrophies.
Aug 11, 2010
Spinal Muscular Atrophy May Also Affect the Heart
Along with skeletal muscles, it may be important to monitor heart function in patients with spinal muscular atrophy (SMA). These are the findings from a study conducted by Nationwide Children’s Hospital and published online ahead of print in Human Molecular Genetics.
Aug 10, 2010
Parent Project Muscular Dystrophy Awards $600K to Nationwide Childrens Hospital for Gene Therapy Study
Parent Project Muscular Dystrophy (PPMD), the largest non-profit organization in the United States focused on finding a cure for Duchenne muscular dystrophy (Duchenne), announced that PPMD will award a $600,000 grant to Nationwide Children’s Hospital in Columbus, Ohio to conduct clinical
Jul 07, 2010
The Ohio State University College of Medicine Department of Pediatrics Ranked 19th in United States
The Ohio State University College of Medicine Department of Pediatrics, housed at Nationwide Children’s Hospital, was ranked 19th on U.S. News & World Report’s list of “Top Medical Schools” among the top pediatric programs at medicals schools in the United States.
Jun 07, 2010
Nationwide Children's Hospital Offers Podcasts Focusing on Neuromuscular Disorders
Scientists and clinicians at Nationwide Children’s Hospital have launched a monthly podcast entitled, “This Month in Muscular Dystrophy,” featuring internationally known scientists discussing the latest research in muscular dystrophy and other neuromuscular disorders.
Mar 01, 2010
New Gene Therapy Study Reverses Effects of Lethal Childhood Muscle Disorder in Mice
Reversing a protein deficiency through gene therapy can correct motor function, restore nerve signals and improve survival in mice that serve as a model for the lethal childhood disorder spinal muscular atrophy, new research shows.
Jan 13, 2010
Investigators Identify Gene Mutations that Predispose Patients with Becker Muscular Dystrophy to Early Onset Cardiomyopathy
Investigators in The Research Institute at Nationwide Children’s Hospital have identified a link between specific modifications of the dystrophin gene and the age of cardiac disease onset in patients with Becker muscular dystrophy (BMD).
Nov 11, 2009
Treatment to Improve Degenerating Muscle Gains Strength
A study appearing in Science Translational Medicine puts scientists one step closer to clinical trials to test a gene delivery strategy to improve muscle mass and function in patients with certain degenerative muscle disorders.
Mar 06, 2006
Passive Injury Prevention Strategies Credited with Decrease in Baby Walker-Related Injuries
EMBARGOED FOR RELEASE: March 6, 2006 Researchers from the Center for Injury Research and Policy at Columbus Children’s Hospital have shown that mobile infant walker-related injuries decreased 76% between 1990 and 2001, thanks to the use of passive injury prevention strategies, such as baby
Aug 30, 2005
Columbus Children's Hospital Researchers Prove Vagus Nerve Stimulation (VNS) is Effective in Treating Children With Epilepsy
Epilepsy, a brain disorder that occurs when the electrical signals in the brain are disrupted, affects about two million Americans. Most people with Epilepsy respond well to medication, but in approximately 25-30 percent of patients, medication doesn't work.
Jul 13, 2005
The Dangers of Mini Trampolines and Full-Sized Trampolines are Similar
Prior research has already proven the dangers of recreational full-sized trampoline use, but now a new study from researchers at Columbus Children’s Research Institute on the campus of Columbus Children’s Hospital has proven that injury patterns associated with mini trampolines
Jun 09, 2005
Vaccine Being Developed to Prevent Ear Infections in Children
Hoping to prevent ear infections for the more than 15 million children in the United States who suffer from them, a promising new vaccine candidate to prevent middle ear infections (otitis media) is being developed by researchers at the Columbus Children’s Research Institute (CCRI) on the
May 03, 2005
Columbus Children's Hospital Leads First National Muscular Dystrophy Newborn Screening Study
Columbus Children’s Research Institute (CCRI) at Columbus Children’s Hospital recently received a grant from the Centers for Disease Control to develop and implement a newborn screening trial for Duchenne muscular dystrophy, the most common type of muscular dystrophy in children.
Jan 01, 0001
Investigators Develop Technique to Effectively Edit NK Cells to Target Specific Cancer Cells
Originally, NK cells seemed promising for a natural therapy to fight cancer due to their innate ability to recognize any cells expressing stress or signs of infection. But investigators quickly realized they faced three key challenges: first, it was difficult to grow a large enough number of NK cells in the lab to offer useful infusions at a reasonable cost; second, an ideal universal donor solution was needed to make NK cell products faster, more reliably and with off-the-shelf availability; and third, NK cells were resistant to genetic modifications that might help them better target specific types of cancer cells. Nationwide Children’s Hospital investigators and their collaborators painstakingly solved the first two problems over the past decade. Now, they’ve conquered the third.
Jan 01, 0001
A Single-Dose Cancer Immunotherapy via Gene Transfer
Researchers at Nationwide Children’s Hospital report proof-of-principle results for a new gene therapy cancer treatment. The off-the-shelf, single-dose immunotherapy serves as an alternative to CAR-T therapy and can be engineered to be on-demand.