'We don’t have time': Parents of 4-year-old with rare disorder urge FDA to reconsider drug denial

The FDA denied approving DCA to treat a rare disorder called PDCD. The Higbees are urging the agency to reconsider.
FOX 8 News on MSN

Rare disease causing meat allergy sees ‘explosive rise’ across US: study

A once-rare disease that causes a person to develop a severe allergy to red meat is seeing an "explosive rise" across America ...
Open Access Government

MHRA to fast-track rare disease treatments in major reform

Explore recent changes in rare disease treatments regulation. Discover how these reforms aim to enhance patient access in the ...
STAT

Parents of children with rare diseases ask: How long until our CRISPR miracle?

Lozano is a rare disease mom, neuroscience Ph.D. candidate at UC Davis, and board member for the PURA Syndrome Foundation. In May, a historic moment in science and medicine was captured in a single ...
American Lung Association

Spotting a Rare Lung Disease in Primary Care

PAP is a rare lung condition that can be difficult to diagnose because it may mimic many other more common lung diseases,” ...
Medscape

Sclerosing Mesenteritis: What GIs Need to Know About This Rare Disease

The American Gastroenterological Association (AGA) has issued an updated pragmatic review on sclerosing mesenteritis (SM). Published in Clinical Gastroenterology and Hepatology, the update evaluates ...
STAT

Rare disease patients caught in Trump crackdown on foreign grant awards

Megan Molteni reports on discoveries from the frontiers of genomic medicine, neuroscience, and reproductive tech. She joined STAT in 2021 after covering health and science at WIRED. You can reach ...
Medscape

Rare Eye Disease Gets First NHS Drug Recommendation

The National Institute for Health and Care Excellence (NICE) has issued final draft guidance recommending idebenone (Raxone, Chiesi Pharmaceuticals) for treating visual impairment from Leber ...
Hosted on MSN

Child Infected with Measles Dies from Rare Complications Years Later: ‘A Painful Reminder of How Dangerous Measles Can Be’

The "school-aged" child developed a rare but fatal disease after recovering from measles A "school-aged" child developed a deadly disease years after recovering from a case of the measles that they ...
FierceBiotech

Chiesi fertilizes Arbor's rare disease gene therapy efforts with $2B biobucks deal

A few months after launching its lead gene therapy into the clinic, Arbor Biotechnologies has found a European partner to come along for the ride. Italian pharma Chiesi is fertilizing Arbor’s rare ...