Rare Diseases
FDA Rejection Derails PTC Therapeutics’ Rare Disease Hopeful
PTC Therapeutics; FDA rejection; vatiquinone; Friedreich’s ataxia; rare disease; efficacy concerns; complete response letter; clinical trial failure
Diagonal Therapeutics Secures $128 Million Series A Funding through Innovative Antibody Agonists
Diagonal Therapeutics, Series A funding, Antibody agonism, Cellular “on” switches, Rare diseases