July 9, 2026 Apertura Gene Therapy Announces Cooperative Research and Development Agreement with Institutes of the NIH to Test Investigational Gene Therapy for Niemann-Pick Disease Type C1 — Cooperative Research and Development Agreement (CRADA) will utilize Apertura’s TfR1 CapX™ Capsid — — Ara Parseghian Medical Research Fund, a non-profit organization dedicated to finding a treatment …
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Parseghian Foundation
Studying Rare Diseases Can Yield Advances For All Of Us
August 11, 2019 • By Elsa Partan & Heather Goldstone • Living Lab Radio on WCAI-FM, Cape Cod, MA Listen to the entire radio interview here: It’s tough to study rare diseases. Because they affect only a small percentage of the population, it can be hard for researchers to find funding. It’s also challenging to …