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Neil McFarlane, president and CEO of Zevra Therapeutics, discusses the rare lysosomal storage disorder Niemann Pick disease type C, the FDA’s upcoming decision on whether to approve the drug, and its broader efforts to build itself into a rare disease therapeutics company. @globalgenes #RARECast

Giles Platford, president of the Plasma-Derived Therapies Business Unit at Takeda, discusses its work in plasma-derived therapies, its recently approved therapy for the rare neuromuscular condition CIDP, and what issues need to be addressed to ensure an adequate supply of human plasma for therapeutic applications.

Salvador Rico, chief medical officer of Encoded Therapeutics, discusses the company’s lead program in Dravet syndrome, its efforts to develop gene therapies with optimized regulatory elements to target specific organs, and why he believes its approach is a point of differentiation for the company.

Steve Roberds, chief scientific officer of the TSC Alliance, discusses the organization's success with crafting a research agenda, how it’s been able to invest in ways that catalyze research, and what it’s done to facilitate drug development by industry.

Sunitha Malepati, founder of the Buffalo Initiative, discusses how she grew frustrated with the drug development landscape, how the Buffalo Initiative plans to fund patient advocacy organizations drug development efforts, and what the initiative is doing to reduce the time and cost of developing a therapy.

Frederic Revah, CEO of the non-profit Genethon, discusses the limits of licensing out its discoveries to biopharma, the different development strategies it pursues, and how it determines the best path for a particular development program.