Sana Biotechnology, CAMP4 Therapeutics and Crinetics Pharmaceuticals have all used the third quarter to refocus their ...

Learn more about whether BioMarin Pharmaceutical Inc. or Ultragenyx Pharmaceutical Inc. is a better investment based on ...

Learn more about whether Ultragenyx Pharmaceutical Inc. or Viridian Therapeutics, Inc. is a better investment based on AAII's A+ Investor grades, which compare both companies' key financial metrics.

"Spinogenix’s ALS hopeful to advance to registrational trial" was originally created and published by Clinical Trials Arena, ...

Mother Daphne Graskewicz-Prado of Southern Illinois told Newsweek: “I wasn’t going to accept the terminal diagnosis.” ...

Announced positive results from two pivotal Phase 3 Essential3 studies of ulixacaltamide HCl in essential tremor (ET); Pre-NDA meeting with FDA ...

Sarepta nevertheless plans to push for full FDA approval of Vyondys 53 and Amondys 45 based on what it said are “encouraging ...

Roche may have been exploring ways to shuttle drugs to the brain for well over a decade, but that’s not stopping the pharma from ...

Third quarter total revenue of $160 million, Crysvita® revenue of $112 million and Dojolvi® revenue of $24 millionReaffirm 2025 Revenue Guidance: ...

Ultragenyx Pharmaceutical RARE reported third-quarter 2025 loss of $1.81 per share, wider than the Zacks Consensus Estimate ...

The UK regulator is creating a new framework for how it will approve rare disease treatments, which it intends to publish in ...