California’s recent decision to create a Rare Disease Advisory Council is a milestone that brings new hope to people living ...

Rare conditions present their own difficulties in terms of medicines adherence, but HCPs, pharma and patients can pool their experience to develop effective, tailored support. It is well ...

Guest Opinion. About 7,000 identified rare diseases impact approximately 1 in 10 Oklahomans. The Cherokee Nation's first Rare ...

Ayik is an advocate for people living with rare diseases. Recently, the smoldering embers of conflict in my family’s rare disease community have burst into flames over the use of an eponym ...

Ibuprofen counters problems caused by mutations in the MAN1B1 gene, fruit fly tests show. Early results in three children are ”fairly positive.” ...

Pitts, a former FDA associate commissioner, is president of the Center for Medicine in the Public Interest and a visiting professor at the University of Paris School of Medicine. Over the summer ...

The University of Minnesota’s annual Rare Disease Day seminar this year, held at the end of February, explained how patients with rare diseases can utilize telehealth services nationally and ...

Boulder County health officials said people can catch tularemia through bites from infected insects, most commonly ticks and ...

CDER Director Patrizia Cavazzoni and CBER Director Peter Marks provided an update on the rare disease innovation hub in a new FDA Voices blog post. FDA’s Patrizia Cavazzoni, director, Center for Drug ...

Patient engagement is a necessity when performing health technology assessment to help better assess and identify the outcomes of a rare disease.

Rare Pediatric Disease Designation is granted by the FDA for serious or ... All statements, other than statements related to present facts or current conditions or of historical facts, contained in ...

Tallulah Moon, 5, has been diagnosed with SPG56, a degenerative brain disease that is caused by a rare gene mutation ... one in every million children, statistics show. "We'd gone from watching ...