Walid Kamoun, Vice President, Global Head of R&D Oncology at Servier, shared a post on LinkedIn:
“While my focus is usually on oncology, stepping into another therapeutic space offers valuable insights into patient experiences, development challenges, and regulatory expectations. It becomes even more rewarding when I get to learn from the senior author Karaa Amel who also happens to be my wife and an expert in this domain.
I want to highlight a new article, “Primary mitochondrial diseases: A rare disease virtual workshop,” which distills key insights from the Reagan‑Udall Foundation for the FDA and FDA virtual workshop held on May 22, 2025. The paper synthesizes discussions among regulators, industry, clinicians, scientists, patients, families, and advocacy groups on how to optimize clinical development for PMD from improving trial enrollment to sharpening endpoint selection, outcome measures, patient population definitions, and statistical strategies that align with regulatory standards.
Congratulations to all authors.
Title: Primary mitochondrial diseases: A rare disease virtual workshop
Authors: Catherine Pilgrim-Grayson, Anna Choe, Naomi Knoble, Yan Wang, Kerry Jo Lee, Lea Ann Browning‑McNee, Cartier Esham, Amel Karaa.
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