“Bubble boy disease” was once a death sentence. A scientific breakthrough changed that. By Simar Bajaj A common cold was enough to kill Cora Oakley. Born in Morristown, N.J., with virtually no immune ...
LOS ANGELES--(BUSINESS WIRE)--Rarity PBC, a Public Benefit Corporation dedicated to expanding the availability of transformative gene therapies for rare diseases, today announced the closing of a $4.6 ...
Overall and event-free survival of 100 and 95%, respectively, seen with autologous CD34+ hematopoietic stem-cell lentiviral gene therapy. HealthDay News — Autologous CD34+ hematopoietic stem-cell ...
An investigational gene therapy showed sustained clinical efficacy for children born with ADA-SCID. In a study of 62 children, overall survival after treatment was 100% and event-free survival was 95% ...
A one-time gene therapy using a patient’s own stem cells has effectively cured a deadly immune disorder in 95% of treated children, offering a lasting, donor-free solution to ADA-SCID, known as the ...
A blood stem cell gene therapy co-developed by UCLA’s Dr. Donald Kohn restored immune function in 59 of 62 children with ADA-SCID, a rare and fatal immune disorder, with no serious complications ...
Two months after she was born, Eliana Nachem got a cough that wouldn’t go away. Three weeks later, she also started having runny stool, prompting a visit to her pediatrician. Eliana didn’t have ...
If you cannot download a file from the Internet or save or run it using Firefox, Chrome, Edge, Opera, or other browsers on Windows 11/10; that is, when you click a link to download a file, nothing ...
Musician Portable has turned himself in at the State Criminal Investigation Department (SCID) in the Yaba area of Lagos. Our correspondent confirmed the development from the spokesman of Lagos Police ...
Researchers headed by teams at Newcastle University, the Wellcome Sanger Institute, and the Great North Children’s Hospital have identified a single gene mutation common to 11 pediatric patients with ...
Your support goes further this holiday season. When you buy an annual membership or give a one-time contribution, we’ll give a membership to someone who can’t afford access. It’s a simple way for you ...
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