The FDA’s first Platform Technology Designation marks a major step forward for rare disease treatments, allowing reuse of key therapy components across related conditions. This breakthrough could accelerate CMD therapies, but Cure CMD needs your support to keep the momentum going.

Modalis Therapeutics program for LAMA2-RD gene therapy granted Rare Pediatric Disease Designation by FDA.

Long-awaited publication about the Phase 1 Open-Label Study of Omigapil in Patients With LAMA2- or COL6-Related Dystrophy now published.

Please see the end of this article for Frequently Asked Questions - CALLISTO Phase 1 Clinical Trial Pratteln, Switzerland, April 5, 2018...