Intrathecal onasemnogene abeparvovec improved HFMSE scores in patients with SMA type 2, indicating enhanced motor function. The STEER study confirmed the therapy's favorable safety profile, with ...
Kerala's Health Department extends free Risdiplam supply to Spinal Muscular Atrophy patients up to 25 years, broadening the previous limit of 12 years. This initiative under the CARE program, launched ...
In a multidisciplinary follow-up study, researchers analyzed bone health decline and the effects of nusinersen in patients with spinal muscular atrophy (SMA) types 2 and 3. SMA is a progressive, ...
We were unable to process your request. Please try again later. If you continue to have this issue please contact customerservice@slackinc.com. Topline results from a phase 3 clinical trial showed an ...
"Decreasing AMMO [active maximal mouth opening] over time is seen most often in patients with SMA type 2 with an estimated reduction of almost 1 mm per year...At this point, we propose that ...
Some patients with later-onset spinal muscular atrophy (SMA) type 2 and type 3 had improved motor function when the investigational monoclonal antibody apitegromab was added to their treatment, the ...
PHILADELPHIA--(BUSINESS WIRE)--Exegenesis Bio, a rapidly growing global genetic medicines company, is pleased to announce the presentation of clinical efficacy and safety data from its EXG001-307 ...
A new study found Spinraza treatment restores a key amino acid called taurine in SMA type 1, which may improve clinical ...