MRI in McArdle Disease (GSDV)

计划入组 87 人 · 首次发布于 2019 年 2 月 18 日

Oral Ketone Body Supplementation in Patients With McArdle Disease

计划入组 8 人 · 首次发布于 2019 年 5 月 10 日

McArdle Disease Treatment by Ketogenic Diet

计划入组 16 人 · 首次发布于 2020 年 3 月 3 日

liver disease program

siRNA therapeutics for ocular disease/dermatological disease (Phio Pharmaceuticals)

undisclosed drug (fibrotic liver disease/NASH), Engitix/Takeda

Rare Disease Research, LLC

CASTLEMAN DISEASE COLLABORATIVE NETWORK

United States

CURE RARE DISEASE, INC.

United States

Edgewise Therapeutics Expands EDG-5506 Clinical Program with New Duchenne Trial for Gene Therapy Recipients

New Alzheimer's Treatments Slow Disease Progression, Offer Hope for Early-Stage Patients

FDA Approves Zanvastro, First Disease-Modifying Therapy for Alexander Disease