A mother has taken to social media to share her daughter's battle with a rare genetic condition that causes her to smell ...
New gene therapy for maple syrup urine disease shows promise in preventing deadly symptoms in cows and offers hope for patients.
A study led by UMass Chan researchers demonstrated that a gene therapy to correct a mutation that causes maple syrup urine disease (MSUD) prevented newborn death, normalized growth, restored ...
For Rare Disease Day, a mum named Cheyenne has taken to social media to spread awareness to her daughter's rare but serious ...
In honor of Rare Disease Day, a mom has taken to social media to spread awareness of her daughter rare disease which caused ...