New SMA Europe documentary aims to raise awareness on research

SMA Europe has released a new documentary titled “One Community. Shared Dreams.” as part of an awareness initiative aimed at highlighting the importance of interdisciplinary collaboration among researchers to advance the understanding of spinal muscular atrophy (SMA). The documentary shares the personal stories of people around the…

Cure SMA Details Its ‘Clinical Trial Readiness Program’

A readiness program to help clinical trial sites prepare to run spinal muscular atrophy (SMA) studies has been established by Cure SMA in collaboration with the pharmaceutical industry, other patient advocacy groups, and research institutes. The program is described in a position statement, “The SMA Clinical…

In UK and Ireland, SMA Patients Demand Reimbursement for Spinraza

A drug appraisal committee of Britain’s National Institute for Health and Care Excellence (NICE) met in Manchester, England, for the third and final time March 6 to decide whether to recommend Spinraza (nusinersen) for the treatment of spinal muscular atrophy (SMA). Several hundred demonstrators organized by the nonprofit group…