Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization ...

Charcot-Marie-Tooth disease tends to go unnoticed for several years before it is confirmed as a diagnosis. Read more at ...

The Haslam family knew early on that their 10-year-old and 12-year-old were suffering from muscular dystrophy, but what they didn't know is that one day, the University of New Mexico Hospital would ...

Few fields in medicine are as promising and complex as gene therapy. As researchers race to develop cures for previously ...

Exceeds the Upper End of Guidance Range for Full-Year 2024 with Unaudited Global Revenues of Approximately $363.7 Million-- --2025 Global ARIKAYCE Revenues Expected to be Between $405 Million ...

What's on the horizon for the life sciences industry? Robin Johnson, PharmD, Gene Therapy Submissions Lead, inSeption Group What unique challenges do gene therapy submissions face compared to ...

Press Release Distributed by ABNewswire.com To view the original version on ABNewswire visit: Netherton Syndrome Treatment Market Size in the 7MM was ~USD 25 Million in 2022, is projected to increase ...

Professor Michel Sadelain, of Canadian origins, was announced the 2025 laureate for King Faisal Prize in Medicine for his groundbreaking contributions to Chimeric Antigen Receptor (CAR) T-cell therapy ...

UHealth and Holtz Children’s Hospital physicians are exploring a new gene therapy called ELEVIDYS to treat older Duchenne muscular dystrophy (DMD) patients. The therapy delivers a functional ...

Amid recent backlash stemming from market withdrawals and trial delays, the FDA seeks to further clarify its requirements for ...

Welcome to This Day in History! Today, we take a journey through time, exploring significant events that have shaped the ...

German regulatory agency, the Paul Ehrlich Institute, granted approval to expand the INVISE Phase 1 clinical trial evaluating INT2104 for the ...