In the time since the Novartis gene therapy Zolgensma was approved for babies with spinal muscular atrophy, other treatments ...
A Prescription Drug User Fee Act (PDUFA) target action date for apitegromab has been set for Sept. 22, 2025.
A rare genetic condition, Spinal Muscular Atrophy (SMA) affects one in 7,000 people in India. It mostly affects children and ...
To Novartis, fresh late-stage data support the idea that its intrathecal drug, which has the same active ingredient as ...
Speakers from the Muscular Dystrophy Association (MDA) highlighted 75 years of progress in understanding and treating ...
Since 2016, the FDA has approved three disease-modifying treatments for spinal muscular atrophy, with several ...
Results from the Phase III STEER and Phase IIIb STRENGTH trials show that Novartis’ investigational intrathecal gene therapy onasemnogene abeparvovec ... in patients aged 2 to <18 years with spinal ...
A pair of clinical data sets gives Novartis confidence in a new formulation of the company’s gene therapy Zolgensma for the ...
Gene therapy, with its offer of a possible cure for rare diseases like sickle cell, is losing early investors to ...
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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 ...
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Mayo Clinic Q&A: Understanding carrier screening for family planningMy husband and I want to expand our family and are trying to get pregnant with our first baby. I’ve read about family ...
Gene therapy, which promises a possible cure for rare diseases like sickle cell, is losing early investors to higher-reward ...
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