Understanding a patient’s motivation for treatment is the key for a physical therapist to empower that patient for the best outcome. For a patient with Duchenne muscular dystrophy, a […]
PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended the renewal of the conditional marketing authorization of Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients five years and older. In connection with the renewal, the marketing authorization will include a specific obligation to conduct an additional long-term post-authorization trial.
Capricor Therapeutics, Inc. (NASDAQ: CAPR), a clinical-stage biotechnology company developing first-in-class biological therapies for cardiac and other serious medical conditions, today announced that it intends to expand its CAP-1002 clinical development program in Duchenne muscular dystrophy (DMD) to encompass the skeletal muscle aspects of the disease, in addition to the cardiac complications.
Our Journey Deaing with Duchenne Our son Joshua was born in 2007. Joshua, our oldest son, is an amazing and fun-loving boy. He was diagnosed with Duchenne Muscular Dystrophy in 2013. […]
Thank you to Marathon Pharmaceuticals for providing updated information about deflazacort access and regulatory status during a webinar this week. The webinar addressed Marathon’s Expanded Access Program for deflazacort and […]
Approach: Anti-fibrotic
Funding Impact: Accelerated identification of lead compound and de-risked pre-clinical program
This informational webinar highlighted the regulatory process to date, updated the community on Marathon Pharmaceutical’s Expanded Access Program (EAP) for deflazacort and discussed what a potential FDA approval of deflazacort would mean in terms of access to the drug in the United States. Some of those who import versions of deflazacort from abroad may have received an update from their distributor with initial details, which was discussed along with steps deflazacort patients might choose to take to help ensure continued access to U.S. supply of deflazacort if FDA approval is granted.
Approach: Anti-inflammatory, anti-osteoporosis, potential steroid sparing therapies
Funding Impact: Industry partnership to sponsor clinical trial for Duchenne
Approach: Cardiac stem cell derived therapy
Funding Impact: Initiation of Phase 1/2 study (HOPE Clinical Trial)
Successful Exit: Stock Return
