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FDA Announces first Platform Technology Designation: Implications for CMD’s Path to Clinical Trials
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.
Gustavo Dziewczapolski
Jun 273 min read


One Step Closer to LAMA2-RD Clinical Trials: Modalis Therapeutics
Modalis Therapeutics program for LAMA2-RD gene therapy granted Rare Pediatric Disease Designation by FDA.
Gustavo Dziewczapolski
Oct 1, 20242 min read


Omigapil Publication Now Available
Long-awaited publication about the Phase 1 Open-Label Study of Omigapil in Patients With LAMA2- or COL6-Related Dystrophy now published.
Rachel Alvarez
Jun 5, 20242 min read


Santhera Announces Successful Completion of First Clinical Trial with Omigapil in Patients with Cong
Please see the end of this article for Frequently Asked Questions - CALLISTO Phase 1 Clinical Trial Pratteln, Switzerland, April 5, 2018...
Cure CMD
Apr 4, 201811 min read
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