RFK Jr.'s Health Department shut down an advisory group that recommends how to test newborns for genetic disorders.

The raise will go toward trialing the company’s lead drug for phosphomannomutase-2 congenital disorder of glycosylation, a ...

The biotech is developing an experimental drug for the most common of a family of disorders all caused by errors in ...

A curved penis because of Peyronie’s disease can be a little embarrassing ... you probably don't need treatment. If sex is hard or impossible, medicines, injections, and sometimes surgery ...

With the safety of gene therapies thrown into the spotlight by a series of FDA clinical holds, Astellas has said that an interim readout in a phase 1/2 trial of its Pompe disease candidate AT845 ...

American depositary receipts of Argenx rose after the company received approval from the Food and Drug Administration for a new delivery method of its treatment for some patients with certain ...

placebo-controlled Phase 3 trial evaluating the safety and efficacy of atacicept in patients with IgAN who have persistent proteinuria and remain at high risk of disease progression. Participants ...

Although the exact mechanism of action of inebilizumab-cdon remains unknown, clinical data have shown significant benefits in reducing disease activity. The US Food and Drug Administration (FDA) has ...

The study's lead author, Florey Deputy Director Professor Clare Parish, Head of Stem Cells and Neural Development, said Parkinson's disease is ... is an emerging treatment to replace those dead ...

"Treatment for mitochondrial diseases within reach." ScienceDaily. www.sciencedaily.com / releases / 2025 / 04 / 250409114701.htm (accessed April 22, 2025). Explore More ...

Vyvgart Hytrulo improved functional and QoL scores in gMG and CIDP with no rise in infections over nine treatment cycles ... demonstrating sustained disease control of generalized myasthenia ...

Koeberl previously invented a gene therapy limited to treating late-onset Pompe disease that was licensed to AskBio. For nearly two decades, Dr. Koeberl has been working toward a treatment that ...