Remove Disease Remove FDA Approval Remove Treatment
article thumbnail

FDA Approves Duvystat, New Oral Treatment for Duchenne Muscular Dystrophy (DMD)

PLOS: DNA Science

A new drug has entered the arsenal against Duchenne muscular dystrophy (DMD), a genetic disease that affects boys and is challenging to treat. FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. In 2023, two gene-based treatments became available.

article thumbnail

University Hospitals now offering FDA-approved medication for treatment of Alzheimer’s disease

SCIENMAG: Medicine & Health

CLEVELAND—University Hospitals (UH) Brain Health & Memory Center is now treating patients with LEQEMBI® (lecanemab), a Food and Drug Administration-approved medication for the treatment of Alzheimer’s disease.

Insiders

Sign Up for our Newsletter

This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.

article thumbnail

Binghamton researchers get FDA approval for drug to treat world’s most common genetic disease

SCIENMAG: Medicine & Health

. — A new drug developed by professors from the School of Pharmacy and Pharmaceutical Sciences at Binghamton University has received Food and Drug Administration (FDA) approval for the treatment of patients with Duchenne muscular dystrophy (DMD), a common genetic disease that mostly affects young boys.

article thumbnail

FDA Approves Landmark Sickle Cell Gene Therapies, Casgevy and Lyfgenia

Drugs.com

Food and Drug Administration on Friday approved two milestone gene therapies for sickle cell disease, including the first treatment ever approved that uses gene-editing technology. FRIDAY, Dec. 8, 2023 -- The U.S. Casgevy, developed by Vertex.

article thumbnail

FDA approves new treatment for Pompe disease

The Pharma Data

Food and Drug Administration approved Nexviazyme (avalglucosidase alfa-ngpt) for intravenous infusion to treat patients 1 year of age and older with late-onset Pompe disease. “Pompe disease is a rare genetic disease that causes premature death and has a debilitating effect on people’s lives,” said Janet Maynard, M.D.,

article thumbnail

Bile acids and the microbiome: revolutionising disease approaches

Drug Target Review

The study’s findings offer new insights into the biochemical language microbes use to influence distant organ systems, which could revolutionise the way researchers approach disease. For example, bile acid treatments have been effective against SARS-Cov-2 infections. Bile acids and bile activated receptors in the treatment of Covid-19.

Disease 111
article thumbnail

FDA Approves Omvoh for Moderately to Severely Active Crohn Disease

Drugs.com

Food and Drug Administration has approved Omvoh (mirikizumab-mrkz) for the treatment of moderately to severely active Crohn disease in adults, according to a press release from Eli Lilly and Company.Omvoh targets. THURSDAY, Jan. 16, 2025 -- The U.S.