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Rare Diseases

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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

Health News

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



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

Health News

Treatment for rare esophagus disease deemed breakthrough therapy by FDA

Health Stories

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

Health Stories

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

Health Stories

Penn doctor makes research strides into his own rare disease

Carroll - Dr. David Fajgenbaum

Health Stories

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


Health News

Rare, flesh-eating STD diagnosed in England


Health News

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

Stock_Carroll - Philadelphia University Jefferson

Health Stories

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



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

Health News

Wharton MBA grad leading fight against rare disease - his own


Health News

Utah teenager has rare water allergy


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