Biogen Medical Research
Rare Diseases
Scientific Objectives
The following areas of research are currently being considered:
- Friedreich’s Ataxia (FA):
- Biological, clinical, and patient-reported measures of disease – natural history and impacts to progression with modifying treatment
- Biological, clinical, and patient-reported measures of disease – natural history and impacts to progression with modifying treatment
- Spinal Muscular Atrophy (SMA):
- Real-world experience of nusinersen after gene therapy in pediatric patients with available neurofilament, motor function, swallowing/feeding, and quality of life data (retrospective analyses of multicenter cohorts)
- Real-world experience (motor function) of adult patients treated with nusinersen following risdiplam treatment (retrospective analyses that must include trajectory data on risdiplam prior to switching therapy)
- Real-world experience (neurofilament + motor function) of gene therapy-naïve pediatric patients treated with nusinersen following risdiplam treatment (retrospective analyses that must include trajectory data on risdiplam prior to switching therapy)
The following areas of research are currently not being considered:
- Amyotrophic Lateral Sclerosis (ALS)
Research support is awarded on a highly competitive basis, and submission of research proposals in these areas of interest does not guarantee support will be awarded.