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Collaborations That Transform: Rare Disease Spinal Muscular Atrophy
Dian Donnai: The rise of rare diseases: partnerships in practice
Discovery Matters | Ep30. Oligos part 2
Coping with the Challenge of Rare Disease: 25 Years of Lessons Learned with Dr. Al Freedman
Risdiplam and Spinal Muscular Atrophy
Discovering Zolgensma for SMA: What goes into making the most expensive drug on the market?
Accredo's Patient Centric Approach to Rare Conditions
Success stories of advanced therapies
Local company develops therapies for rare diseases
Disease Modifying Therapies | Spinal Muscular Atrophy 2023
Stoke Therapeutics: Working on Rare Genetic Diseases
Rare Motor Neuron Disease
