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
As a parent of a son with Duchenne I have been frustrated that the data presented to the FDA does not match the patient experience. I personally know so […]
The Collaborative Trajectory Analysis Project (cTAP), a public-private partnership to accelerate data science solutions to critical problems in drug development for Duchenne Muscular Dystrophy (Duchenne), today announced the publication of two research studies with important implications for the design of effective clinical trials.
Every Duchenne parent wants to know that their child is in good hands with their medical team. Physical therapy is an important part of that medical team, and Texas physical […]
NEWPORT BEACH, CA. October 25, 2016 – CureDuchenne, a nonprofit dedicated to funding research and improving patient care for Duchenne muscular dystrophy, will host an informational webinar on November 1, […]
Consortium Working Together to Bring Quality of Life Treatments to the Duchenne Community NEWPORT BEACH, Calif., October 25, 2016 – The next wave in tackling Duchenne Muscular Dystrophy is getting […]
A physical therapist is required to take on multiple roles while caring for their patients, and this is especially true when treating a complex condition such as Duchenne muscular […]
