Rare disease advocacy group urges Trump administration to restore FDA clarity

A rare disease advocacy coalition on Wednesday urged the ‌Trump administration to restore regulatory clarity as it considers ...

US FDA warns of liver injury cases tied to Amgen's rare disease drug

By Puyaan Singh March 31 (Reuters) - The U.S. Food and Drug Administration on Tuesday said it has identified cases of liver ...

Biogen to buy Apellis Pharmaceuticals for $5.6 billion in rare disease push

March 31 (Reuters) - Biogen has agreed to acquire Apellis Pharmaceuticals for about $5.6 billion in cash, expanding its ...

Inside Actor Dudley Moore's Lonely Final Days, After Being Diagnosed With a Rare Brain Disease

The British actor, who won an Oscar for 1982's 'Arthur,' was diagnosed with a brain disease in 1998 and died in New Jersey ...
Medscape

Rare Sexual Disorders in Men May Challenge Diagnosis

Limited evidence and overlapping symptoms contribute to diagnostic uncertainty in rare sexual disorders, highlighting the ...

They're in a race to save their children with a rare disease. A newly approved drug has given them hope.

After a string of denials for rare disease treatments, the FDA greenlit a drug for Hunter syndrome, a progressive illness ...

Biogen uplifts rare disease pipeline with $5.6bn Apellis buyout

Biogen has forged a $5.6bn deal to acquire immunology and rare disease specialist Apellis Pharmaceuticals – marking another M ...
BioSpace

Biopharma M&A Heats Up, Rare Diseases Win Three Approvals, Wave Crashes

Biogen, Eli Lilly, Merck and Novartis spent more than $20 billion to absorb biotechs with promising or approved drugs; the ...
BioSpace

FDA Ties Amgen’s Rare Inflammatory Disease Drug to Eight Deaths, Dozens of Liver Injuries

The FDA in January asked Amgen to pull Tavneos from the market, citing liver toxicity issues that affected the drug’s overall ...

Changing the long search for rare disease diagnoses with new AI breakthrough

A newly developed AI tool can dramatically speed up the search for the genetic causes of rare diseases, a process that often ...

Wellesley MA 'Plunge for Elodie' fights rare skin disease

The ninth annual 'Plunge for Elodie' in Wellesley will raise funds and awareness for epidermolysis bullosa, a rare skin disease.

North Carolina girl with rare Sanfilippo Syndrome loses access to promising treatment after clinical trial ends

Sadie has been fighting since birth. She underwent two brain surgeries and spent 73 days in the neonatal intensive care unit ...