Jensen Caraballo, a beloved disability rights advocate in New York, has died at age 33. Born with spinal muscular atrophy, ...

Affordable generic version of SMA drug Risdiplam soon available in India, offering hope to patients and activists.

Despite significant advancements in spinal muscular atrophy (SMA) management, unmet needs persist, especially for patients ...

Multidisciplinary coordination across prescribing teams, nursing, laboratory medicine, finance, and infusion centers is crucial for gene therapy delivery in Duchenne muscular dystrophy (DMD).

for apitegromab for the improvement of motor function in patients with spinal muscular atrophy (SMA). Apitegromab, a fully human monoclonal antibody, works by selectively binding to the pro- and ...

Apitegromab is an investigational treatment aimed at improving motor function for people living with spinal muscular atrophy. Scholar Rock said it has also submitted and received validation for its ...

The NIH is considering a catastrophic cut to indirect cost reimbursements for public universities, which would hurt the ...

22. Apitegromab is an investigational treatment aimed at improving motor function for people living with spinal muscular atrophy. Scholar Rock said it has also submitted and received validation ...

Results from the STEER and STRENGTH studies showed that OAV101 IT led to a 2.39-point improvement in motor function in patients with spinal muscular atrophy. Results from the Phase III STEER and Phase ...