Spinal muscular atrophy
In the latest results shared by the company, treatment with nusinersen for a median of 4.9 years helped participants maintain and make progressive gains in motor function.
Novartis released new data Monday from its Phase III SPR1NT trial that reinforces the benefits of Zolgensma.
Biohaven Pharmaceutical Holding announced the acquisition of Channel Bio and new licensing rights to a spinal muscle atrophy drug from Bristol Myers Squibb.
Biogen exercised an option to pick up an exclusive global license for a drug to treat spinal muscular atrophy from Ionis. The compound, BIIB115, is an antisense oligonucleotide.
Voyager’s primary focus will be on developing gene therapies for Huntington’s disease, Amyotrophic Lateral Sclerosis (ALS), and pre-clinical research in spinal muscular atrophy and diseases linked to GBA1 mutations.
The FDA-initiated partial hold on Novartis’ gene therapy trial for spinal muscular atrophy has been lifted recently. Here’s everything you need to know about it.
New data shows Zolgensma, Novartis’ gene therapy for spinal muscular atrophy, has the potential to be used presymptomatically in juveniles.
After discovering promising indicators on the path to the development of Branaplam (LMIO70) for spinal muscular atrophy, Novartis now hopes to repurpose the drug for the treatment of Hungtington’s disease.
Biogen recently announced plans to initiate a Phase IV clinical trial to determine the benefit of Spinraza in patients who received Zolgensma. The two therapies have markedly different ways of treating the disease.
Genentech, a Roche company, announced the U.S. Food and Drug Administration (FDA) had approved its Evrysdi (risdiplam) for spinal muscular atrophy (SMA) in adults and children two months of age and older.
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