In the time since the Novartis gene therapy Zolgensma was approved for babies with spinal muscular atrophy, other treatments ...
To Novartis, fresh late-stage data support the idea that its intrathecal drug, which has the same active ingredient as ...
Posters presented at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference show that therapeutic ...
Intrathecal onasemnogene abeparvovec (OAV101 IT), an investigational, one-time gene replacement therapy, demonstrated a ...
A pair of clinical data sets gives Novartis confidence in a new formulation of the company’s gene therapy Zolgensma for the ...
Novartis also shared results from the open-label phase 3b STRENGTH study of OAV101 IT in SMA patients aged two to less than 18 years who had discontinued treatment with nusinersen or risdiplam, which ...
Novartis (NVS) announced positive safety and efficacy results from the Phase III program for investigational intrathecal onasemnogene ...
MedPage Today on MSN2d
Young Patient Dies After Receiving New Gene Therapy for Duchenne Muscular DystrophyA young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy ...
Spinal muscular atrophy type 1 is a genetic disorder that typically appears within the first six months of life, causing ...
Scholar Rock will present new Phase 3 SAPPHIRE trial data on apitegromab at the 2025 MDA conference, highlighting efficacy ...
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