The gains of scientific advancements in treatment are shadowed by the cuts coming in Medicaid coverage at rare disease fair.

Patients, doctors and legislators spoke about the struggles of those with rare diseases.

Breast cancer is the most common form of cancer in the UK but is rare in younger women. Women in their 20s accounted for only ...

Trey Robertson has long struggled with sickle cell but has tried not to let that slow him down — and he encourages others to ...

YOU’D be forgiven for thinking in this day and age you’d be safe from catching some of the grim diseases that blighted the ...

The Cherokee Nation held its third annual Rare Disease Summit to advance policy, care, and early intervention initiatives for ...

Zilurgisertib came through by way of a $16 million licensing deal struck between Mirum and Incyte earlier this year. (monticelllo/iStock/Getty Images Plus) In just ...

BARCELONA, Spain — For children living with rare respiratory conditions, fighting a disease involves two battles. “The first is the physical illness,” said Natalia Maeva, patient representative from ...

The neurological deterioration and progressive muscle weakness caused by Alexander disease now have their first FDA-approved therapy, a genetic medicine that Ionis Pharmaceuticals designed to address ...

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When Dustin Matteson moved his family to Orange County’s idyllic Ladera Ranch community in 2011, at first, it seemed like the perfect place to raise a child. Its green, open spaces and ...

Through sheer determination, Neena Nizar has become the first patient in a clinical trial testing a treatment for her disease — one so rare it affects just 30 people worldwide. By Gina Kolata Neena ...