The FDA approved copper histidinate (Zycubo) as the first treatment for Menkes disease in pediatric patients, the agency ...

Gene therapy treatments for rare diseases are being developed, but getting them out of the lab has proved challenging.

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

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

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

ANI Pharmaceuticals (NASDAQ:ANIP) outlined plans to accelerate its shift toward rare disease medicines while maintaining profitability and cash generation from its generics business, according to ...

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

Rare diseases are defined as conditions affecting fewer than 200,000 people in the US or less than 1 in 2,000 in Europe. 1-2 While each disease is individually rare, collectively, they represent a ...

Rare diseases - a medical condition so obscure that even your doctor has to Google. Despite their name, these diseases collectively impact millions of Americans, creating a healthcare system that’s ...

Rare growth disorders are a diverse group of uncommon conditions that affect an individual’s physical development. They may stem from genetic mutations, hormonal imbalances, or other complex factors.

More than 500 European patients with previously undiagnosed rare disorders received a genetic diagnosis through research carried out by an extensive European collaboration, the Solve-Rare Diseases ...

Amongst the political shockwaves that have to date characterized President Donald Trump’s first few weeks back in the Oval Office, it is likely to be the proposed changes to medical research funding ...