News

Stem Cell Therapy May Benefit Some Type 1 Infants

Treatment with human fat tissue-derived mesenchymal stem cells (MSCs) may improve motor nerve cell health and survival in infants with spinal muscular atrophy (SMA) type 1, according to data from a small Phase 1 clinical trial in Iran. Notably, the stem cell treatment appeared to have effectively slowed disease progression…

Texas Adds SMA to Its Newborn Screening Program

Newborn screening for spinal muscular atrophy (SMA) is now offered to 85% of all annual births in the U.S., Cure SMA has announced. The milestone, which means that eight in 10 babies are getting tested at birth, was reached after Texas included the newborn screening on June 1,…

Major Developmental Milestones Met in Infants, Zolgensma Data Show

New data about Zolgensma, the one-time gene therapy for children with spinal muscular atrophy (SMA), demonstrate age-appropriate development when used pre-symptomatically, and rapid, meaningful efficacy in symptomatic children, even those with severe disease before treatment, according to two Phase 3 clinical trials. The therapy, developed by Novartis, uses…

EveryLife Introduces First of Kind ‘Roadmap’ to ICD Codes

To help patient advocacy leaders and their partners better understand how global health statistics codes — known as ICD codes — are assigned, updated, and revised in the U.S. health information system, the EveryLife Foundation for Rare Diseases is presenting a first-of-its-kind resource guide. The foundation created the…

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