A study led by UCL found that individuals with rare diseases were five times more likely to die from Covid-19 than the ...

Kristina Krassman is battling two diseases that cause extreme pain in her stomach and abdomen when she tries to eat.

D.C.’s Vanda Pharmaceuticals Inc. has locked in a deal with a West Coast biotech to commercialize a drug for a rare skin ...

Addison’s disease is a rare autoimmune disorder where the adrenal glands don’t produce enough vital hormones, causing fatigue ...

DEBRA Research and LEO Pharma enter strategic partnership to advance treatments for rare skin diseases: Munich, Germany Thursday, February 6, 2025, 10:00 Hrs [IST] DEBRA Research ...

The FDA has made some modest efforts, but it needs an internal organization that understands and would be an aggressive advocate for the development of rare-disease drugs. When I joined the FDA to be ...

Epidermolysis Bullosa (EB), or butterfly disease, affects 1 in 50,000 people, causing fragile skin that blisters easily, often likened to the delicacy of butterfly wings.

Navigating the regulatory landscape for clinical trials, particularly for rare diseases like celiac disease, presents a unique set of challenges. The complexities arise from both the intricate ...

New Delhi: Finally, there is some hope for patients suffering from rare and life-threatening skin disorders, as the existing national policy for rare diseases, which currently covers 63 conditions ...

Mulikat Okanwalon campaigns for victims of Noma, an infection that causes facial disfigurement | ITV News Meridian ...

In two new papers, researchers from The Jackson Laboratory (JAX) report the successful use of two approaches—gene therapy and ...