Spinal muscular atrophy (SMA) is a genetic neuromuscular disease affecting specialized nerve cells that control voluntary muscle movement, according to the Muscular Dystrophy Association (MDA). It can ...
Please provide your email address to receive an email when new articles are posted on . At the time of last follow up, 82.6% of children born with SMA type 1 from 2016 on were still alive. Of the 140 ...
Patients with SMA often suffer from impaired bulbar function, which negatively affects quality of life and can be difficult to treat effectively. A German study prospectively followed children with ...
Oral risdiplam (Evrysdi, Genentech) started in the first 6 weeks of life let most infants with presymptomatic spinal muscular atrophy (SMA) reach motor milestones typical of healthy babies, results of ...
A single-dose gene replacement therapy is found to improve movement ability in children over 2 years of age and teenagers with spinal muscular atrophy, according to research published in Nature ...
Sidra Medicine Administers Qatar's First Itvisma Gene Therapy For Spinal Muscular Atrophy. The Peninsula One-time gene ...
A new clinical trial has revealed encouraging results for a muscle-targeting therapy aimed at improving motor function in children and adolescents with spinal muscular atrophy, according to a study ...
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'It doesn't really allow us enough time': Ralston family scrambling after Children's Nebraska ends care program
A Ralston family has weeks to find new care for their son with spinal muscular atrophy before school starts after Children's ...
Researchers evaluated oxygen saturation and arterial CO2 variables to screen for sleep disordered breathing in children with SMA type 1-3. Of the measures evaluated, only an oxygen desaturation index ...
The fundraiser that was launched to help nine-month-old baby Matthias with Spinal Muscular Atrophy (SMA) receive urgent gene therapy has closed after reaching its goal. Checks by Mothership on Jul. 31 ...
Biogen Inc. (NASDAQ:BIIB) on Wednesday announced in a press release the topline results from the Phase 1 study of salanersen (BIIB115/ION306), an antisense oligonucleotide (ASO) being developed for ...
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