Rare Diseases

Drug Development

CRISPR and other new technologies open doors for drug development, but which diseases get prioritized?

Health Insurance

Independence bolsters access to gene-based therapies used to treat rare, inherited health conditions

IBX Gene Therapy

Celine Dion's stiff person syndrome diagnosis brings attention to a rare disease

CHOP patient with spinal muscular atrophy to walk the runway ahead of New York Fashion Week

SMA Mom

Monkeypox 'very unlikely' to cause a COVID-like outbreak, scientists say

Treatment for rare esophagus disease deemed breakthrough therapy by FDA

Rare disease researcher offers unique perspective on search for COVID-19 treatment

COVID-19 drug inventory

Children's Health

Kawasaki disease may be a COVID-19 complication for some young children

Kawasaki disease and COVID-19

His life in 'overtime,' Penn doctor races to find better treatments for rare Castleman disease

David Fajgenbaum Castleman Disease book 2

Flesh-eating bacteria are in warm coastal waters – but it doesn't mean you'll get sick

Flesh-eating bacteria

Penn doctor makes research strides into his own rare disease

Carroll - Dr. David Fajgenbaum

Collegeville boy, 6, gets wish to become pilot for a day

09072018_William_Beyer_Cockpit

Rare, flesh-eating STD diagnosed in England

07202019_sex_unsplash

Jefferson Health researchers identify cause of skin cancer in Butterfly Syndrome patients

Stock_Carroll - Philadelphia University Jefferson

North Jersey family, CHOP doctor discuss journey to discover boy's rare disease on 'Today' show

Elijah_Patino_Neil_Romberg_CHOP

How a drugmaker turned the abortion pill into a rare-disease profit machine

Pills on a counter

Wolf pushes for a Rare Disease Advisory Council in Pennsylvania

Wharton MBA grad leading fight against rare disease - his own

Utah teenager has rare water allergy

041715_waterallergy

Just In

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