Solid Biosciences receives orphan drug designation from FDA and EMA for Duchenne Muscular Dystrophy

Release Date: 26-Nov-2016

Solid Bioscience and its subsidiaries declares that Food and Drug Administration and European Medicine Agency has granted orphan drug designation to company’s gene therapy candidate, SGT-001, for the treatment of patients with Duchenne muscular dystrophy (DMD). 


Duchenne muscular dystrophy (DMD) is a very rare and progressive muscle damaging disease affecting approximately 1 in 3,500-5,000 boys born worldwide and is the most common fatal genetic disorder diagnosed in childhood. The disease is mainly caused by the absence of dystrophin protein, an important mediator between cardiac and skeletal muscle function. The gene therapy is developed or designed to protect and maintain functional dystrophin expression in the muscles.  


The FDA Orphan Drug Designation is granted to medicines and biologics that are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment medicine.


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