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 ...

Findings from St. Jude Children's Research Hospital demonstrate that virtual memory T cells, a specialized group of immune cells, provide nonspecific immunity for infants early in life. The work stems ...

A senior lecturer at Prince Abubakar Audu University (PAAU), Anyigba, Kogi, Dr Olabode Abimbola Ibikunle, has passed on while allegedly having sexual affair with a female student. The incident ...

The world's top chess player quit a major tournament in New York City after officials told him he couldn't wear jeans to compete. The 2024 events began on Thursday. Carlsen arrived at Friday's events ...

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 ...

A child prodigy has set a new record in the chess world by besting a grandmaster nearly 30 years older than him on Sunday. At just 8-years-old, Singaporean chess prodigy Ashwath Kaushik became the ...

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 ...