Neurogene has a $903M market cap with two phase 1/2 assets, NGN-401 for Rett Syndrome and NGN-101 for CLN5 Batten Disease.

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

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

The Hamilton scientists were able to map a CWD prion, the misfolded protein that causes the deadly brain disease, to the near-atomic level.

For many people, filling out paperwork about their family's health history is a routine part of a medical visit. For adopted people like Ramya Gruneisen, it's impossible.

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 ... This might include certain cancers, heart disease or dementia. But it might also raise ethical questions about ...

Nearly a quarter of patients with hidradenitis suppurativa (HS) experienced concomitant eye disease, and among those who did ...

Roughly one out of every ten people in the United States are affected by rare diseases. Many of these conditions are life‑threatening, and most ...

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

Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) (the “Company” or “Quoin”), a clinical-stage, specialty pharmaceutical company focused on developing and commercializing novel treatments for rare and orphan ...