Max Farmer was born healthy but then diagnosed with a rare and genetic condition that causes progressive muscle wasting when ...

Representatives of Russian patent unions have sent an appeal to the Russian Scientific Center for Expertise of Medical ...

The global antisense oligonucleotides market is poised for remarkable growth over the next decade, projected to rise from USD 2,913.5 million in 2023 to USD 5,519.6 million by 2033 moving forward with ...

including the EU following approval by the European Commission (EC); additional global submissions in progress to expand WAINUA access SPINRAZA ® (nusinersen) for the treatment of spinal muscular ...

Ionis’ pipeline of antisense drugs is beginning to produce results – a separate alliance with Biogen resulted in development and approval of Spinraza (nusinersen), approved for spinal muscular ...

Cost-effectiveness body NICE has rejected Biogen’s Spinraza (nusinersen) therapy for the rare genetic muscle wasting disease spinal muscular atrophy (SMA) because of its high cost – but is in ...

Alongside Skyclarys, Biogen’s rare disease business is headed by spinal muscular atrophy treatment Spinraza (nusinersen). Spinraza generated global Q1 2025 sales of $423.9m, up from $341.3m in Q1 2024 ...

Harrison Gabel and Steven Gray Kathie Bishop Renowned leaders in neuroscience, gene therapy, and translational research join ...

- On track for second independent launch with donidalorsen PDUFA August 21, 2025 - - Increasing 2025 financial guidance by more than 20% - CARLSBAD, Calif.--(BUSINESS WIRE)-- Ionis Pharmaceuticals ...