Praxis Precision Medicines develops therapies for central nervous system disorders, with candidates targeting depression and ...

Two new drug applications (NDA) for ulixacaltamide in essential tremor (ET) and for relutrigine in SCN2A and SCN8A developmental and epileptic encephalopathies (DEEs) have been submitted to the U.S.

Ultragenyx Pharmaceutical's employees were already braced for changes as the biopharma considered how to rebound from a pair ...

Pharm Exec’s 21st Annual Pipeline Report examines emerging drug development classes, treatment modalities, and expansion ...

Pharm Exec’s 21st Annual Pipeline Report examines emerging drug development classes, treatment modalities, and expansion pursuits in five therapeutic settings ripe for next-gen innovation.

Biotech dealmaking is increasingly defined by a clear strategic shift: Pharmaceutical companies are prioritizing de-risked, ...

Even when promising clinical trials exist, and the FDA allows expanded access, rare disease patients like my granddaughter ...

Dr. Maarten Dewilde, an associate professor at KU Leuven, sat down with Technology Networks to discuss molecular engineering approaches that have proven effective in improving the transport of ...

Ultragenyx Pharmaceutical RARE reported fourth-quarter 2025 loss of $1.29 per share, wider than the Zacks Consensus Estimate ...

Ultragenyx recently resubmitted a BLA seeking approval for the gene therapy after the FDA rejected it, and the firm will resubmit it again in response to new questions.

CD Bioparticles launches specialized RNA Synthesis services for researchers to develop next-generation mRNA vaccines, gene therapies, and RNA-based therapeutics.

Pages Latest Report] According to a market research study published by Custom Market Insights, the demand analysis of Global Amyotrophic Lateral Sclerosis Therapeutics Market size & share revenue was ...