Cautiously good news for the Tucson-based national Muscular Dystrophy Association : a new drug could help stop devastating muscle deterioration in certain patients.
The association announced Wednesday that an experimental drug for patients with Duchenne Muscular Dystrophy is moving into clinical trials with humans.
Duchenne Muscular Dystrophy, the most common kind of Muscular Dystrophy among children, can result in cognitive deficits, an inability to walk and a short life span because of weakened cardiac and respiratory muscles.
The drug SMC C1100 is an orphan drug meant to compensate for dystrophin, the protein missing among Duchenne patients, who are overwhelmingly male.
The national association says its Venture Philanthropy drug program supported the development of the drug with a $750,000 grant to British-based Summit PLC, a pharmaceutical discovery company.
The association also gave $1.8 million to geneticist Kay Davies at Oxford University for laboratory studies related to compensating for dystrophin loss.
Patients with Duchenne Muscular Dystrophy are unable to make dystrophin, resulting in muscle fiber degeneration.
The protein utrophin produced with the help of the drug can restore dystrophin and researchers anticipate it will overcome the deficit in Duchenne patients by restoring and maintaining the function of muscles, including the heart and diaphragm.