While we are thrilled to share a recent press release from Modalis Therapuetics , it’s important to keep in mind that the information provided includes “forward‑looking statements.” That means the company is sharing what they hope or plan to do, but these steps are not guaranteed and can change. The scientific paper mentioned in the press release describes years of careful testing of Modalis’ experimental therapy for LAMA2 (called MDL‑101) in animal models. Publishing this work is an...

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.

From shaping care guidelines in the Netherlands to tackling gene therapy challenges at the MDA Summit, Cure CMD is driving progress in research and care. Join us at the 2025 SciFam Conference this August in Philadelphia—registration is open!