Risdiplam continues to show promise as a therapy for individuals with spinal muscular atrophy (SMA) types 2 and 3, results from an ongoing Phase 2/3 trial show. Results from the study, “Update from SUNFISH Part 1: Safety, Tolerability and PK/PD from the Dose-Finding Study, Including…
Risdiplam (RG7916) can improve both survival and motor function in patients with type 1 spinal muscular atrophy (SMA) beyond that expected in the natural history of the disease, according to recent clinical data. These data were presented during the 2019 annual meeting of the American Academy of…
The European Medicines Agency has granted a priority medicines, or PRIME, designation to oral therapy candidate risdiplam to treat spinal muscular atrophy (SMA). PRIME designation is intended to encourage the development of promising therapies by speeding their evaluation so that the therapies, if proven effective, can be available to patients…
An increased amount of SMN protein — crucial to the survival of motor neurons — in the blood could reflect similar increases in the brain, spinal cord, and peripheral tissues after spinal muscular atrophy (SMA) treatment with the oral therapy risdiplam, researchers say. They believe these findings in animal…
Preliminary data from two ongoing clinical trials into risdiplam (RG7916) — a potential oral therapy for all types of spinal muscular atrophy — support its considerable promise, with treated babies now able to “roll, sit, kick … things that they [otherwise] never do,” the CEO of PTC Therapeutics said in…
The investigational treatment risdiplam (RG7916) improved motor function in patients with type 1, 2 or 3 spinal muscular atrophy (SMA), according to preliminary results from the first part of two Phase 2/3 clinical studies. Risdiplam, being developed by Roche and Genentech in collaboration with PTC Therapeutics and the …
Infants with spinal muscular atrophy (SMA) who are presymptomatic and given the gene therapy Zolgensma in the first six weeks of life have better motor, respiratory, and nutritional outcomes, according to a recent study from Europe. However, researchers found functional motor scores still improved significantly, albeit less dramatically,…
Oral strength and endurance — important for functions like swallowing, speaking, and chewing — were found diminished in adults with spinal muscular atrophy (SMA) in three out of five assessments, according to a new study. In particular, untreated SMA type 2 patients who couldn’t walk had the most…
Branaplam (also known as LMI070) is an oral small molecule that was being developed as a potential therapy for SMA. In 2021 its development was discontinued based on the fact that the therapy would no longer represent a highly differentiated option for SMA patients.
Newborns diagnosed with spinal muscular atrophy (SMA) who were treated with Evrysdi (risdiplam) before the onset of symptoms are continuing to reach developmental milestones such as sitting, standing, and walking independently, with many achieving them in a time frame expected of typical child development. That’s according to two-year…