Ultra-rare disorders are those affecting fewer than one person in 50,000. Sometimes, indeed, they are unique. And unique ...

Dr Sudheendra Rao N R from the Organization for Rare Diseases India reveals that approximately 70 million are affected by ...

Rare diseases are genetic disorders that affect a small proportion of the population. Despite their rarity, they can be a ...

When Casey McPherson became a father, his purpose became clear. He needed to raise his daughter, Rose, to be loving, courageous, healthy and strong. He needed to protect her. Many parents can ...

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

EAU CLAIRE, Wis. (WEAU) - While most families countdown to Christmas, Erin Binder is counting something else: time. Her 14-year-old twin sons, Grady and Jace, both have PKAN, a rare genetic disorder ...

This article is part of Nature Outlook: Skin, an editorially independent supplement produced with financial support from LEO Pharma. About this content. KaDee Troop is the mother of seven adopted ...

Elamipretide shows promise in treating Barth syndrome. Advocates hope it will win FDA approval. Walker Burger, who has Barth syndrome, injects the drug elamipretide at his home in Monroe on Friday, ...