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

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 ...

A wearable robotic device called HAL improved walking ability for SMA patients who also received Spinraza (nusinersen), a ...

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

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

The paradigm of care is shifting as disease-modifying therapies including nusinersen, onasemnogene abeparvovec and risdiplam alter the disease trajectory of SMA. The objective of this study was to ...

- 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 ...

Spinal muscular atrophy treatment Spinraza (nusinersen) became Biogen’s best-selling drug in Q1 2025. Image credit: Shutterstock/ Tada Images. Biogen has unveiled Q1 2025 revenues above estimates, ...

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 ...

CARLSBAD, Calif.--(BUSINESS WIRE)--Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) (the “Company”) today reported financial results for the first quarter ended March 31, 2025. “With an encouraging start to ...

Motor improvements in adults with spinal muscular atrophy (SMA) treated with Spinraza (nusinersen) paralleled a boost in the strength and duration of electrical impulses along nerves that supply the ...