Everyone’s favorite little blue pill may offer more than just a boost in the bedroom. New research suggests the erectile ...

The FDA proposes new rules allowing mutation-specific gene therapies to qualify for approval despite extremely small patient ...

The move comes months after the Trump administration touted leucovorin as a potential therapy for a broader group of patients with autism symptoms.

As newborn screening and rapid DNA sequencing become routine, we are poised to catch and treat inherited diseases at their earliest stages. Today, we can intervene in the first days or weeks of life.

Stanford Medicine pediatric hematologist Agnieszka Czechowicz, MD, Ph.D., has devoted her research career to improving ...

Adrenal insufficiency encompasses a spectrum of disorders in which the adrenal glands fail to produce adequate amounts of steroid hormones, notably glucocorticoids and, in some cases, ...

Genetic testing can help doctors diagnose some neurological disorders, such as Huntington’s disease. It can also provide insights into a person’s risk of future health conditions. Genetic testing ...

Nick Jonas' neighbour Maya Kibbel recently died of Wilson's disease at the age of 30. The disease is a rare genetic disorder ...

If you haven’t heard of the genetic disorder Heterotaxy, a new children’s book released by a Connecticut teacher and her ...

Canadian Senators Jane Cordy and Marie-Françoise Mégie joined the GANSID CEO, Lanre Tunji-Ajayi, M.S.M and the World Inherited Blood Disorders community in the #TieRed Campaign From Blood drives to ...