MAP THE SMA: a Machine-learning Based Algorithm to Predict THErapeutic Response in Spinal Muscular Atrophy

计划入组 247 人 · 首次发布于 2023 年 3 月 15 日

Economic Impact of Guidelines for Gastroesophageal Reflux Disease

计划入组 484 人 · 首次发布于 2003 年 3 月 28 日

A Study Comparing the Acceptability of Pentasa® Sachets Versus Pentasa® Tablets in Children With Crohn´s Disease

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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

FDA Raises Doubts About Stealth BioTherapeutics' Elamipretide for Barth Syndrome

FDA Approves PTC Therapeutics' Kebilidi, First Direct-to-Brain Gene Therapy

AI Accelerates Clinical Trial Recruitment, Reducing Enrollment Times and Costs