Researchers compared children with types 1 and 2 spinal muscular atrophy (SMA) in terms of body composition and resting energy expenditure, and found those measures — for now — are the best to use when attempting to determine their appropriate dietary needs. Body mass and body weight index Z-scores (a measure…
Nusinersen, an investigational drug for the treatment of spinal muscular atrophy (SMA) is acceptably safe and well tolerated, and shows encouraging clinical efficacy according to a study published in the leading medical journal The Lancet.
Good Start Genetics and Roche Diagnostics have entered into a marketing agreement to sell Good Start’s GeneVu carrier screening service for inherited genetic disorders, such as cystic fibrosis (CF) and spinal muscular atrophy (SMA). Under the terms of the contract, Roche Diagnostics will provide obstetricians and general practitioners in the…
PTC Therapeutics announced the initiation of a Phase 2 clinical trial to test the safety, tolerability, and efficacy of the small molecule RG7916 for the treatment of children and adults with type 2 and type 3 spinal muscular atrophy (SMA).
Blocking molecules that normally act as muscle degrading factors may offer a treatment approach for milder forms of spinal muscular atrophy (SMA), demonstrated in experiments with a mouse model of SMA type 3. The study, “Activin Receptor Type IIB Inhibition Improves Muscle Phenotype and Function in a Mouse Model…
Spotlight Innovation announced that it has acquired an exclusive, worldwide license to commercialize STL-182, an orally administered small molecule under investigation as a potential treatment for spinal muscular atrophy (SMA). SMA is an autosomal recessive disorder. Between 1 in 40 and 1 in 50 adults have only a single intact…
A poor contribution by the ribcage to breathing is a characteristic feature of children with spinal muscular atrophy (SMA) type 1 and 2, and is apparent from infancy. The study, “Spontaneous Breathing Pattern as Respiratory Functional Outcome in Children with Spinal Muscular Atrophy (SMA),” published in the…
Most people would be willing to pay to test their newborn baby for spinal muscular atrophy (SMA) even if no direct treatment is available for the disease, according to a study published in the journal Pediatric Neurology.
Spinraza (nusinersen) for spinal muscular atrophy (SMA) met the primary endpoint in an interim analysis from a Phase 3 clinical trial evaluating the treatment in children with later-onset (consistent with Type 2) SMA. Biogen and Ionis Pharmaceuticals reported that children treated with Spinraza had a highly statistically significant improvement in motor…
The European Medicines Agency (EMA), SMA Europe, and TREAT-NMD network will lead a one-day workshop to discuss, support, and advance development of new therapies to treat spinal muscular atrophy (SMA), a neuromuscular disease. The “Spinal Muscular Atrophy Workshop” will be held 9 a.m. – 7 p.m. at…
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