Two Austin sisters are hopeful after the FDA recently approved two new treatments for their rare genetic disorder, ...

Progeria, or Hutchinson-Gilford Syndrome, comes from Greek and means 'prematurely old'. It is an extremely rare genetic ...

Zevra Therapeutics' drug, Miplyffa, for treating a rare and fatal genetic disorder will have a wholesale acquisition cost ...

The play is known as the Jackson Special. Kelly Maynard, Jackson’s mom, said he was diagnosed with Duchenne’s Muscular ...

The largest and most diverse study to date of epilepsy's genetic factors has revealed new potential targets for treatment, ...

The 56-year-old has a rare genetic disorder called incontinentia pigmenti. The condition causes skin abnormalities like a ...

The U.S. Food and Drug Administration approved IntraBio's drug for a rare and fatal genetic disorder, the health regulator ...

After a seven-year search for answers, a family from Eden, Utah, has received a life-changing diagnosis for their daughter ...

Author: Dr Shrikant Jamdade, Clinical Geneticist, Mumbai Spinal Muscular Atrophy (SMA) is a common genetic disorder affecting ...

Miplyffa treats Niemann-Pick disease type C, which progressively impacts the abilities to speak, swallow, and walk or move ...

An Overland Park teen completed a 150-mile bike journey Sunday to raise awareness and hope for his rare genetic disorder.

Shares of Biohaven climbed 13% premarket on Monday after the biotech company said its treatment for a rare neurological ...