News | March 14, 2026
Biogen unveils promising new data for Salanersen in kids with SMA
Salanersen was generally well-tolerated, slowed neurodegeneration, and enabled functional gains
Salanersen was generally well-tolerated, slowed neurodegeneration, and enabled functional gains
Industry leaders highlighted the pharmaceutical sector’s role in advancing research, innovation, early screening, and multi-stakeholder collaboration
Achondroplasia, a rare genetic disorder, causes skeletal dysplasia and often increases the risk of muscular, neurological, and cardiorespiratory complications
The deal was executed via a merger of Novartis’ wholly owned subsidiary, Ajax Acquisition Sub, Inc., into Avidity
The recommendation is based on new data from continuous safety monitoring of medicines in the EU
The approval marks a major advance in targeted treatment for this rare, chronic autoimmune disease, offering twice-yearly maintenance dosing after two initial loading doses
PBGENE-DMD is designed to permanently correct mutations in the dystrophin gene between exons 45 and 55, the “hot-spot” region affecting roughly 60% of DMD patients
Nxera will take full responsibility for regulatory approvals, clinical development where required, manufacturing and commercialization in the licensed territories
The partnership also sets the stage for broader collaboration on rare disease treatments
Acromegaly, a rare chronic endocrine disorder caused by excess growth hormone, is seeing promising advances in treatment development
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