Spinal muscular atrophy
Novartis’ gene therapy Zolgensma is again under a cloud of doubt after Nature Biotechnology retracted its 2010 article noting “issues” regarding data cited in a key figure in the report.
Novartis announced Wednesday it is temporarily halting dosing in its ongoing Phase IIb VIBRANT-HD trial of branaplam in Huntington’s disease.
Two patients receiving Novartis’ SMA gene therapy Zolgensma have died from acute liver failure, the company reported Thursday.
Extension data from the Phase II TOPAZ trial showed that apitegromab yielded sustained and continued benefits in non-ambulatory patients with types 2 and 3 spinal muscular atrophy (SMA).
As Scholar Rock prepares to move forward with its experimental assets for spinal muscular atrophy, it has terminated 25% of its staff and also announced the loss of its chief medical officer.
Genentech shared new data from its SUNFISH study evaluating the use of Evrysdi for SMA people ages 2 to 25 years diagnosed with Type 2 or Type 3 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.
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