Rare disease found in Berkeley homeless encampment, officials say

Berkeley health officials are urging people to vacate a homeless encampment after an outbreak of a rare disease that can ...
Becker's Hospital Review

FDA approves 1st treatment for rare pediatric disease

FDA approves 1st treatment for Menkes disease, offering a copper therapy that improves survival in affected children.
Fierce Pharma

JPM26: Leo Pharma, hungry for more partnerships, looks to widen reach in rare dermatology diseases

Danish dermatology specialist Leo Pharma and its CEO Christophe Bourdon came to San Francisco to attend the J.P. Morgan ...
BioSpace

Biopharma Momentum To Propel Rare Disease, Cancer, Neuro Forward in 2026

After years stuck in the “doldrums,” the biopharma sector is in a “very good place” heading into the new year, analysts told ...
pharmaphorum

Sentynl gets first FDA approval for rare disease Menkes

The US regulator has approved Sentynl Therapeutics Zycubo (copper histidinate; formerly known as CUTX-101) as a treatment for ...
BioSpace

Sentynl, Fortress Bounce Back With FDA Approval for Rare Pediatric Disease

The FDA previously rejected Zycubo for Menkes disease in October last year, citing issues with the drug’s manufacturing ...

Muskego family helps raise millions for rare childhood disease treatment

A Muskego family helped raise more than $5.5 million to fund early access treatment for children with Sanfilippo syndrome ...
BioProcess International

Senate blocks Trump-supported FDA fast-tracking of rare-disease medicines

The Priority Review Voucher (PRV) program is designed to incentivize rare disease drug development.

Travere shares fall as US FDA extends review of rare kidney disease drug

The U.S. Food and Drug Administration has extended its review for the expanded use of Travere Therapeutics’ drug to treat a ...