Cure Rare Disease
Cure Rare Disease works on 1 rare disease tracked on Trial Friend, including Duchenne Muscular Dystrophy.
Cure Rare Disease is a nonprofit organization that partners with academic researchers and clinicians to develop advanced therapeutics for ultra-rare genetic diseases. The organization gained attention for its N-of-1 CRISPR gene therapy program (CRD-TMH-001) for Duchenne muscular dystrophy developed with UMass Chan Medical School. Tragically, the sole patient in the trial died in November 2022 from an AAV-related immune reaction, and the program status is under review.
Focus areas at Cure Rare Disease
As a rare disease specialist, Cure Rare Disease has active clinical trial programs and drug development efforts across 1 rare diseases, including Duchenne Muscular Dystrophy. These programs may span orphan drug designation, novel therapeutic mechanisms, and precision medicine approaches targeting the underlying causes of each disease.
The clinical trials section below shows all active and recruiting studies sponsored by Cure Rare Disease, sourced live from ClinicalTrials.gov. Each trial includes its current recruitment status, study phase (Phase 1 through Phase 4), conditions under investigation, and the number of active trial sites. The FDA-approved drugs section lists treatments that have received U.S. Food and Drug Administration approval, with brand names, generic names, approval dates, and matched rare disease indications from the openFDA database.
Cure Rare Disease is headquartered in Cambridge, United States. The company dedicates its research and development resources exclusively to rare and orphan diseases, working to bring innovative treatments to patients with conditions that have historically had limited or no treatment options.
Active Clinical Trials
FDA-Approved Drugs
Rare Disease Focus Areas (1)
Diseases targeted by Cure Rare Disease's clinical trial and drug development programs
Patient Resources
Organizations and resources related to Cure Rare Disease's rare disease focus areas
Frequently Asked Questions About Cure Rare Disease
Common questions about Cure Rare Disease's rare disease programs, clinical trials, and treatments.