Data Supports Deramiocel’s Sustained Efficacy and Safety in Treating DMD
Improvements Seen in Multiple Cardiac and Skeletal Endpoints Demonstrating Stabilization of Cardiac and Skeletal Muscle Function Over 3 Years of Treatment
Company on Track to Fully Submit Biologics License Application (BLA) by End of 2024
SAN DIEGO, Oct. 11, 2024 (GLOBE NEWSWIRE) — Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced positive 3-year safety and efficacy results from its ongoing HOPE-2 open label extension (OLE) study for its lead asset, deramiocel, for the treatment of Duchenne muscular dystrophy (DMD). The data was highlighted in a late-breaking poster presentation at the 29th Annual Congress of the World Muscle Society (WMS), October 8-12, 2024 in Prague, Czechia.
When it comes to the treatment of rare diseases like Duchenne muscular dystrophy, any advancements in therapy are cause for celebration. Capricor Therapeutics’ announcement regarding the positive 3-year safety and efficacy results of deramiocel is indeed a significant milestone in the field of medical research. The data presented at the World Muscle Society Congress demonstrates the potential of deramiocel to not only improve cardiac and skeletal muscle function in DMD patients but also to sustain these improvements over an extended period of time.
Patients with DMD face challenges that affect both their physical health and quality of life. The progressive nature of the disease often leads to muscle weakness and deterioration, particularly in the heart and skeletal muscles. However, the promising results from Capricor’s study suggest that deramiocel could offer a viable treatment option for DMD patients, helping to stabilize muscle function and potentially slow down the disease progression.
With Capricor Therapeutics aiming to fully submit the Biologics License Application for deramiocel by the end of 2024, there is hope that this innovative therapy could soon be available to those in need. The potential approval of deramiocel would not only provide a new treatment option for DMD patients but also pave the way for further advancements in the field of rare disease therapeutics.
How this will affect me:
As an individual who may be affected by Duchenne muscular dystrophy, the positive results from Capricor Therapeutics’ study offer hope for a potential new treatment option that could improve muscle function and quality of life. If deramiocel receives regulatory approval, it could have a significant impact on the management of DMD and may provide a more effective therapy for patients like myself.
How this will affect the world:
The development of deramiocel as a treatment for Duchenne muscular dystrophy represents a major advancement in the field of rare disease therapeutics. If approved, this innovative therapy has the potential to not only benefit DMD patients worldwide but also inspire further research and development in the pursuit of effective treatments for other rare diseases. Capricor Therapeutics’ progress with deramiocel could pave the way for new possibilities in the global healthcare landscape.
Conclusion:
The positive 3-year safety and efficacy results of deramiocel presented by Capricor Therapeutics at the World Muscle Society Congress are a testament to the potential impact of innovative therapies in the treatment of rare diseases like Duchenne muscular dystrophy. With continued advancements in medical research and the promise of new treatment options on the horizon, there is hope for a brighter future for patients living with DMD and other rare diseases.
