Amy Mahood’s child MJ, who will be three next month, was diagnosed with 1p36 deletion syndrome when he was aged 14 months.

Hunter syndrome, also called mucopolysaccharidosis II, is an X-linked genetic lysosomal disorder caused by loss-of-function mutations in the IDS gene, encoding iduronate-2-sulfatase (I2S). I2S is a ...

With such a small risk of illness, does this type of embryo selection border on eugenics? By Kwame Anthony Appiah Kwame Anthony Appiah has been The New York Times Magazine’s Ethicist columnist since ...

Antiphospholipid syndrome, also known as APS, is an autoimmune disease that sits at the intersection of inflammation and blood clotting. Antiphospholipid syndrome is best known for increasing the risk ...

Hypermobile Ehlers-Danlos syndrome (hEDS) is one of the most common heritable connective tissue disorders. Early estimates have reported that this genetic disorder affects at least one in 5,000 ...

Feb 9 (Reuters) - Regenxbio (RGNX.O), opens new tab said on Monday the U.S. Food and Drug Administration had declined to approve its gene therapy for a rare disease called Hunter syndrome, citing ...

After facing a delayed decision deadline and a clinical hold, Regenxbio’s Hunter syndrome gene therapy has been rejected by the FDA. In rejecting RGX-121, the FDA raised concerns about its clinical ...

We’re starting to get a handle on the role that genetics plays in the onset of chronic fatigue syndrome, or myalgic encephalomyelitis (ME/CFS). According to the largest study of its kind to date, more ...

The rate of severe infections decreased from 2 infections to 0.2 infections per PYO after treatment with etuvetidigene autotemcel. The Food and Drug Administration (FDA) has approved Waskyra ...

The FDA has approved Waskyra (etuvetidigene autotemcel), the first gene therapy for Wiskott-Aldrich syndrome, a rare and life-threatening immune disorder. Waskyra is indicated for pediatric patients 6 ...

A child with Hunter Syndrome has amazed doctors with his progress after becoming the first person in the world to receive a pioneering gene therapy for the devastating disease. Three-year-old Oliver ...

A California toddler is the first person in the world to receive gene therapy to treat his devastating disease. Three-year-old Oliver Chu was born with a rare, genetic condition called Hunter syndrome ...