Contributor: By loosening standards, the FDA isn't doing rare-disease patients any favors

It's reasonable to assess rare-disease drugs differently. That's why the FDA has been remarkably flexible about these ...
Yahoo

A Doctor Shares the Number-1 Sign You Have This Rare Disorder

Hemophilia is a rare, genetic blood condition ... M.D., a hematologist with the Penn Blood Disorders Center at Penn Medicine, previously explained when discussing the symptoms that indicate you may ...

Omeros Sells Exclusive Global Rights for Drug Zaltenibart to Novo Nordisk

Omeros Corporation (NASDAQ: OMER) is one of the Reddit stocks that will go to the moon. On October 15, Omeros Corporation and ...
Scientific American

Gene Therapy Marks a Turning Point for Rare Skin Diseases

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 ...
Medical Xpress

Meet your worm avatar: How microscopic worms are helping find new drugs for rare diseases

New research from the MRC Laboratory of Medical Sciences (LMS) provides a powerful, scalable method for finding treatments for rare genetic diseases using tiny, transparent worms. Rare diseases may be ...
USA Today

Her 5-year-old has a severe, rare disease. There's a cure, but she can't get it.

Catherine Illingworth, 39, knew something was wrong the moment her son's neurologist walked into the room. Illingworth requested the referral from her son's pediatrician based on instinct. George took ...