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).
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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…
AveXis reported that the planned pivotal clinical trial of its gene therapy candidate AVXS-101 for spinal muscular atrophy (SMA) Type 1 will enroll about 20 patients and will have a single-arm design, with the comparator being the natural disease history. This update follows the Type B meeting Sept. 30 with the…
The U.S. Food and Drug Administration (FDA) has accepted Biogen‘s new drug application for nusinersen, an investigational drug for spinal muscular atrophy (SMA), for priority review. The company also announced that the European Medicines Agency (EMA) validated nusinersen’s marketing authorization application (MAA). If nusinersen is approved, it will become…
Developmental milestones such as sitting unaided, rolling, crawling, standing or walking are rarely, even partially achieved by infants with type 1 spinal muscular atrophy (SMA), confirms a study published in the scientific journal Neuromuscular disorders.
