Craig Martin, CEO of rare disease advocacy group Global Genes, explains the perils and pitfalls to the patient-led revolution around rare disease therapeutics.
Nace, brand director for Rare Disease Advisor (an MM+M sister brand), joins Marc Iskowitz to discuss trends shaping the awareness and commercialization of rare disease products.
Israel Emerges as Global Powerhouse in Rare Disease Research
Lisa Stockman Mauriello and husband Bob Mauriello, who have been leading a high-profile campaign to gain access to Biogen’s tofersen under so-called expanded access, discuss the drugmaker’s decision this week to make the experimental ALS drug available, but not as soon as they had hoped.