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BridgeBio Pharma, Inc. today announced sustained positive results from PROPEL 2, a Phase 2 trial of the investigational therapy infigratinib in children with achondroplasia, demonstrating continued potential best-in-class efficacy and an encouraging safety profile.

Investor enthusiasm and evolving FDA pathways are accelerating rare disease drug development, with ultrarare conditions like MPS II moving into the spotlight.

Baylis Medical Company has announced that President and co-Founder Kris Shah has been named to the Advanced Medical Technology Association’s (AdvaMed) Accel Board of Directors.

Innovation drives speed in early phase drug development. ICON’s Accelerated Pharmaceutical Solutions integrate advanced formulation design, real-time dose flexibility, and cutting-edge CMC expertise to transform complexity into clarity. By uniting science, technology, and strategy, biotechs can move faster, smarter, and with confidence—turning obstacles into opportunities for breakthrough success.

Accel Clinical Services (ACS), the parent company of Accel Research Sites, is excited to announce its acquisition of the assets of AMPLIFY Clinical.

Global Blood Therapeutics, Inc. has awarded approximately $450,000 to U.S. community-based organizations and institutions as part of the company’s 2021 Access to Excellent Care for Sickle Cell Patients (ACCEL) Grant Program.

Vinay Prasad, chief of the FDA’s Center for Biologics Evaluation and Research, is planning to publish a paper this month to outline his office’s thinking on accelerating gene editing reviews.