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Capricor Advances in DMD Therapy With Positive Study Data


Capricor Therapeutics (NASDAQ: CAPR), a biotechnology firm focused on innovative therapies for rare diseases, has announced favorable three-year results from the ongoing HOPE-2 open-label extension (OLE) study on CAP-1002 for Duchenne muscular dystrophy.

Patients treated with CAP-1002 showed sustained improvements in upper limb function and cardiac performance compared to an external dataset from Cincinnati Children’s Hospital Medical Center, indicating potential long-term benefits.

Statistical Significance in Disease Attenuation

The HOPE-2 OLE study revealed a statistically significant reduction in the decline of upper limb function (PUL 2.0) and stabilization of left ventricular ejection fraction among CAP-1002-treated patients over three years. This contrasts sharply with the expected decline observed in untreated DMD patients, suggesting that CAP-1002 can potentially slow disease progression and improve quality of life.

CAP-1002 has demonstrated a well-tolerated safety profile throughout the study. This favorable safety data supports the continued use of CAP-1002 in treating DMD and provides reassurance for patients and healthcare providers about the long-term use of this therapeutic approach.

Strategic Pathway to FDA Approval

Capricor highlighted the significance of these findings in a recent Type-B meeting with the FDA, which could expedite the pathway to the …

Full story available on Benzinga.com

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