Data from the pivotal Phase 2b ReNeu trial demonstrate that patients with NF1-PN achieved deep responses and improvements in ...

Saturday Nov. 16 is Cadasil awareness day, a genetic disorder categorized by stroke, cognitive decline, and dementia.

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

An international team of researchers has provided a genetic diagnosis for 30 individuals whose condition was undiagnosed for ...

Screening newborn babies for these rare diseases involves sequencing their ... having seen it advertised on a poster during an antenatal appointment. She said her daughter Emilia, born the ...

Then when Josie was 2, Zeitzer Johnson recalled reading a newspaper story about a boy in Texas who had a rare disease ...

The Cherokee Nation is hosting a Rare Disease Summit Nov. 21 to address the unique challenges facing tribal citizens with rare medical conditions. The event, organized by Cherokee Nation Health ...

CASGEVY™ approved for the treatment of patients 12 years of age and older with sickle cell disease (SCD) and transfusion-dependent beta ...

About PYRUKYND® (mitapivat) PYRUKYND is a pyruvate kinase activator indicated for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency in the United States, and for the ...

Nomlabofusp program update expected mid-December to include available safety, pharmacokinetic (PK) and frataxin data, as well as available ...