4-aminopyridine Fails to Improve Muscle Function in SMA Type 3 Patients, Small Trial Finds
Treatment with the molecule 4-aminopyridine (4-AP) failed to improve the motor function, endurance, and muscle strength of spinal muscular atrophy (SMA) type 3 patients who are able to walk, according to results from a small Phase 2/3 clinical trial. Findings from...
FDA Grants Rare Pediatric Disease Designation to SRK-015 for SMA
The U.S. Food and Drug Administration (FDA) has granted the designation of rare pediatric disease to SRK-015, an investigational treatment for spinal muscular atrophy (SMA), its developer, Scholar Rock, announced. This designation is given to therapies that are...
Nerve-muscle Communication Molecule May Be SBMA Therapeutic Target, Study Says
Increasing the levels of brain-derived neurotrophic factor (BDNF) — a molecule involved in the communication between nerve cells and muscle — specifically in muscle delayed symptom onset and slowed disease progression in a mouse model of spinal and bulbar muscular...
Muscle and Combo Therapies Likely Next Focus for SMA, Experts Say
Treatment combinations, including future muscle-targeted therapies, are part of what likely lies ahead for spinal muscular atrophy (SMA), according to experts in the SMA community. In separate interviews with SMA News Today, Richard Finkel and Laurent Servais,...
Spinraza-Zolgensma Combination Well-tolerated in Children with SMA Type 1, Study Shows
Combining Spinraza (nusinersen) with the gene therapy Zolgensma (onasemnogene abeparvovec-xioi) is generally well-tolerated and sustains motor improvements in children with spinal muscular atrophy (SMA) type 1, according to a case series study. The data, which...HCP — SMA News Today
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