In the absence of any marketed drugs, investors are set to focus on BEAM's pipeline updates when it reports first-quarter 2025 earnings.

It also develops UX701 that is in Phase 2 clinical trial for the treatment of Wilson disease; and UX053 for the treatment of glycogen storage disease type III. Ultragenyx Pharmaceutical Inc.

therapy for glycogen storage disease 1a. Roche has claimed a CE Mark in the EU for an AI algorithm that can help doctors decide whether patients with chest pain are having a heart attack.

Creatine has gotten a lot of attention as an aid in building muscle and strength, but can cyclists actually benefit?

A thorough nutrition assessment of a child with liver disease is essential. [11] This includes the collection of anthropometric and biochemical data, as well as a clinical, social, and dietary ...

Apr. 22, 2025 — Researchers have identified a potential new way to treat idiopathic pulmonary fibrosis (IPF), a deadly and currently incurable lung disease that affects more than 3 million people ...

Apr. 23, 2025 — A new study finds vision loss fears may deter some patients from cataract surgery, despite it being the only effective treatment. The research underscores the role of doctor ...

Pneumonia in the Shadow of COVID-19: Along Came COVID Infectious disease experts describe ways in which COVID pneumonia differs from community-acquired pneumonia and how COVID vaccines may offer ...

Denali’s candidate is its DNL12 enzyme replacement therapy (ERT) for mucopolysaccharidosis (MPS) IIIA, a form of lysosomal storage disease also known as Sanfilippo syndrome type A, which is in a ...