Dublin Live

Mum abseils down Croke Park for Rare Ireland who gave priceless support after son's diagnosis

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

Motivated by Daughter’s Tragic Death, Robert Horry Embarks on Plan to Help Millions of Americans

Most of you might measure Robert Horry’s impact through his clutch shots or his seven NBA championships, which he won during ...
BioWorld

Anti-tFR1 nanobody-I2S gene therapy corrects Hunter syndrome neuropathy in mice

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 ...
The New York Times

My First Child Has a Genetic Syndrome. Can I Use I.V.F. to Spare My Second?

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 ...
Medical Xpress

Researchers identify genetic variants and patterns associated with hypermobile Ehlers–Danlos syndrome

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 ...
Medical Xpress

Using gene expression in blood to personalize care for antiphospholipid syndrome

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 ...
Reuters

US FDA declines to approve Regenxbio's gene therapy for rare disease

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 ...
FierceBiotech

FDA rejects Regenxbio's gene therapy in Hunter syndrome, leaving CEO 'concerned'

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 ...
New Scientist

Chronic fatigue syndrome seems to have a very strong genetic element

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 ...
Morningstar

Fondazione Telethon Announces FDA approval of Waskyra™ (etuvetidigene autotemcel), a Gene Therapy for the Treatment of Wiskott-Aldrich Syndrome

WAS affects almost exclusively males, with an estimated incidence of 1 in 250,000 live male births. The therapy represents a major scientific and clinical achievement, offering new hope for patients ...
Monthly Prescribing Reference

Gene Therapy Waskyra Approved for Wiskott-Aldrich Syndrome

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 ...
Becker's Hospital Review

FDA approves 1st gene therapy for Wiskott-Aldrich syndrome

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 ...