We think Biogen's neurology, immunology, and focus on rare diseases support a narrow moat. Biogen's strategy has its roots in the 2003 merger of Biogen (Avonex for treating multiple sclerosis, or MS) ...

The Paris gold medallist on finding her sport, advocating for wheelchair access and how she and her husband make each other ...

The 5 mg risdiplam tablet provides the same efficacy and safety for spinal muscular atrophy as the currently available oral ...

Vincent Gaynor remembers, almost to the minute, when he realized his part in birthing the breakthrough gene therapy Zolgensma ...

Over the past nine years, patients with spinal muscular atrophy have seen the number of FDA-approved treatment options for ...

About Evrysdi Evrysdi is an SMN2-splicing modifier designed to increase functional SMN protein production. With the introduction of a new tablet formulation, Evrysdi now offers an alternative ...

Roche (RHHBY) announced that the Food and Drug Administration has approved a New Drug Application for an Evrysdi tablet for people living with ...

Spinraza treatment does not lead to significant kidney dysfunction in SMA patients with types 1 and 2, a safety and efficacy ...

Liver issues, including a buildup of fat in the liver, occur before the onset of neuromuscular disease, per a study in a mouse model of SMA.

Infants with SMA type 1 born since 2016 experience better functional outcomes and life expectancy with disease-modifying treatments.

A new electrical stimulation therapy for spinal muscle atrophy (SMA) has shown promise in reactivating motor neurons and ...

New research suggests that electrical spinal cord stimulation could improve muscle function for patients who have spinal ...