We must understand that while rare diseases, by definition, affect a small portion of the population, they collectively ...

Public health groups are challenging Roche's attempt to block a generic version of its expensive Spinal Muscular Atrophy drug ...

Israeli researchers discover that better glycemic control can improve overall health of the brain and slow age-related ...

Perspectives of patients with spinal muscular atrophy (SMA) and their caregivers should be weighed before making decisions around SMA-enhancing treatments.

Astellas confirmed today that it will withdraw its EMA filing for avacincaptad pegol as a treatment for eye disorder ...

Gene therapy for Spinal Muscular Atrophy (SMA) is prohibitively expensive in India, with the life-saving drug Zolgensma ...

Astellas Pharma Inc. has withdrawn its marketing authorization application from the EMA for its avacincaptad pegol intravitreal solution for the treatment of geographic atrophy secondary to ...

PulseSight plans to submit a phase I clinical trial authorization (CTA) by the end the year, to be closely followed by a phase II proof-of-concept to demonstrate the efficacy and the safety of its ...