by BNS Staff | Jan 26, 2021 | Just Published, News, Pulmonologist News, slider
Spinraza (nusinersen) effectively preserves respiratory muscle strength in addition to motor function in children with spinal muscular atrophy (SMA) type 2, according to a real-life, small study in France. Larger studies are needed to confirm these findings, and to...
by BNS Staff | Dec 10, 2020 | Just Published, slider
Olesoxime, a small molecule once considered a potential treatment, failed to slow motor and respiratory decline in people with spinal muscular atrophy (SMA) types 2 and 3 who were unable to walk, according to final results from an extension Phase 2 clinical trial....
by BNS Staff | Oct 8, 2020 | Just Published, News, Orthopedics News, slider
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...
by BNS Staff | Sep 30, 2020 | Just Published, News, Orthopedics News, Pediatricians News, Physical Therapists News, slider
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...
by BNS Staff | Sep 24, 2020 | Genetic Counselors News, HCP, Just Published, Neurology News, Orthopedics News, slider
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...
by BNS Staff | Sep 10, 2020 | Just Published, Neurology News, News, Orthopedics News, Pediatricians News, slider
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,...