How an Ultra-Rare Disease Patient Organization Drove Research to a Treatment

An Ultra-Rare Disease Drug Developer Tries to Navigate Regulatory Uncertainty

When Stealth Biotherapeutics sought approval for its experimental therapy to treat the ultra-rare and life-threatening condition Barth […]

Powering Weakened and Stressed Cells in ALS to Function Better with Nanocrystal Therapies

Developing a New Class of Therapies Based on a Natural Cargo Carrier

Reaching Beyond the Limits of Enzyme Replacement Therapies with Gene Therapies

One Woman’s Journey as a Caregiver to a Husband with Frontotemporal Dementia

Treating Rare Endocrine Disorders with Therapeutic Peptides

Tackling the Pricing Challenges for Advanced Therapies for Rare Diseases