Medical Professional Publications

Innovation and Discovery

Phase 1 Study Shows Promise of Gene Replacement Therapy for Spinal Muscular Atrophy Type 1

Children with spinal muscular atrophy 1 (SMA1) who received a single high dose of gene therapy are living longer and have a better quality of life, a study in the New England Journal of Medicine shows. Treated children sat up, rolled over, walked and talked, reaching motor milestones not previously observed in the disease, says Jerry Mendell, MD, principal investigator in the Center for Gene Therapy at Nationwide Children’s Hospital and study leader. SMA1 is the most common genetic cause of infant mortality. The researchers found that the earlier the treatment is given, the greater the impact on the child. The results, they say, add to arguments for including SMA in the Recommended Uniform Screening Panel for newborns.  

Read more in this Pediatrics Nationwide article.

Debunking Misinformation about MiraLAX, Gluten-Free Diets and PPI’s

Misinformation about gluten-free diets, proton pump inhibitors and the key ingredient of MiraLAX and similar laxatives abound on the internet and in published reports. These inaccuracies and falsehoods can become an impediment to treatment and good health, says Carlo Di Lorenzo, MD, chief of the Division of Gastroenterology, Hepatology and Nutrition at Nationwide Children’s Hospital. Di Lorenzo offers statistics, facts and findings from peer-reviewed studies to debunk the “fake news,” as well as alternatives and cautions to consider, in an American Academy of Pediatrics presentation.  

Read more in this Pediatrics Online article.

New Findings Advance Pursuit of an RSV Vaccine

Respiratory syncytial virus, commonly known as RSV, is the leading cause of bronchiolitis and pneumonia in infants in the United States and a leading cause of infant death worldwide. A team of researchers at Nationwide Children’s Hospital working toward a vaccine have found virus genotypes linked to the highest disease severity and discovered that severe RSV makes babies sicker by suppressing the immune system. In another avenue of research, the team found that maternal antibodies in children less than 2 months old appear to be protective and that pre-fusion antibodies block RSV from infecting cells, making them a potential focus for treatment. The team’s studies are published in The Journal of Infectious Diseases.

Read more in this Pediatrics Nationwide article.

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