Skip to main content

Rare Disease

Rare Disease

Development of a novel, phosphospecific alpha-synuclein antibody as a therapeutic for MSA​

Novel Mechanism to Treat Neurodegenerative Diseases

Best In Class Ferroptosis Inhibitors in Friedrich's Ataxia

Brain Endothelium-Targeted Therapy for Cerebral Cavernous Malformations

Innovative approach to treat ALS using a novel small molecule target

Small molecule and enzymatic approaches to revert aberrant phase transitions in neurological disorders

CurIOS: Novel mRNA Therapeutics for Virally-driven Cancers

Development of a New Class of Therapeutics to Treat Poisoning Diseases

Identification of small molecules tuning phase transitions to treat neurological disorders

Rosevix (Rosziqumab) A ROS1 Biologic for Invasive Cancers

Subscribe to Rare Disease