Biogen Medical Research
Rare Diseases
Scientific Objectives
The following areas of research are currently being considered:
Friedreich Ataxia (FA)
- Biological, clinical, and patient-reported measures of Friedreich’s Ataxia in the context of natural history
- Impact of omaveloxolone on daily functioning
- New methods and technological approaches to facilitate the diagnosis of FA
Spinal Muscular Atrophy (SMA)
The following areas of research are being considered from countries where high dose nusinersen (50 mg/5mL and 28mg/5mL) has been approved.
- Real-world treatment outcomes, preferences, and satisfaction/experiences with high dose nusinersen (50 mg/5 mL and 28 mg/5 mL) in treatment-experienced pediatric patients transitioning from nusinersen (12 mg/5 mL), switching from risdiplam, or adding-on after OA
- Primary outcomes of interest include motor function, electrophysiology, ADLs, fatigability, eating/swallowing/feeding, and voice quality
- Proposals that include the evaluation of outcome data prior to switching will be prioritized
The following are additional areas of research in SMA being considered
- Development and validation of outcome measures (including composite measures) and their link to clinical manifestations
Amyotrophic Lateral Sclerosis (ALS)
We are not currently accepting proposals
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.