British mum Laura Mattinson was devastated to learn her one-year-old son Ezra had been diagnosed with Leigh syndrome after he ...

Chris Kirkpatrick is in awe that his former ‘NSync bandmate Justin Timberlake performed a full tour amid a battle with Lyme ...

The U.S. Food and Drug Administration has approved Empaveli (pegcetacoplan) as the first treatment for C3 glomerulopathy (C3G ...

For many rare disease patients and their families, this often results in years of uncertainty, misdiagnosis, and limited or ...

The primary safety risks associated with gene therapy include immune reactions and off-target effects in unintended organs, such as the liver, lungs, and spleen. Nevertheless, Ali and Townsend note ...

Key Points GAAP revenue surpassed expectations, reaching $12.5 million in Q2 2025, Revenue reached $12.5 million, up approximately 45.3% from the prior year period. Net loss (GAAP) widened to $112.0 ...

Biotech firm aims to streamline operations and reinvest in high-growth areas such as oncology and rare diseases as it targets ...

Justin Timberlake has revealed that he has Lyme disease, joining celebrities like Shania Twain, Avril Lavigne, Justin Bieber, ...

Dr. Michael Wagner covers the basics of epithelioid hemangioendothelioma, a rare vascular cancer, spanning symptoms, ...

Sanofi is handing over $130 million upfront for the China rights to Arrowhead Pharmaceuticals’ rare metabolic disease ...

A stellar launch of the rare heart disease med Amvuttra has boosted Alnylam’s market cap above $50 billion. | A stellar launch of Amvuttra in the rare heart disease ATTR-CM has boosted Alnylam’s ...

A mother has discovered her baby has a rare, incurable disease after doctors joked he only had 'happy baby syndrome'. Laura ...