Rett syndrome is a rare developmental disorder, most often due to a mutation in the MECP2 gene on the X chromosome, but it’s not usually inherited. Advances in genetics have launched a new era in ...
Neurogene Gene Therapy Shows Signs of Efficacy in Small Study, But an Adverse Event Spooks Investors
A Neurogene gene therapy in development for the rare neurodevelopmental disorder Rett syndrome has encouraging early data in a small group of patients, an interim readout that comes a week after a ...
In the comfort of her Miami home, 34-year-old Julianne Espinosa lies in bed captivated by videos of Disney princesses flashing across her television screen. As echoes of “The Little Mermaid” fill her ...
SPEIGLETOWN, N.Y. (NEWS10) — Families across the Capital Region came together on Saturday to raise money and awareness for a rare neurological disorder called Rett Syndrome. Saturday was the Reverse ...
Gene therapy may offer hope for people with a rare childhood disease called Rett syndrome. Around one in 10,000 girls are born each year with Rett syndrome, a rare genetic condition that impacts brain ...
We were unable to process your request. Please try again later. If you continue to have this issue please contact [email protected]. Health Canada has approved Daybue to treat Rett syndrome ...
New discoveries about the severe developmental disorder known as Rett syndrome could open the door to better treatments for the devastating, life-shortening condition. Scientists investigating the ...
Rett syndrome is a rare disorder linked to autism that affects brain development. Microglia, the resident immune cells of the central nervous system, have been implicated in the development of Rett ...
The neurodevelomental disease Rett syndrome is usually caused by mutations in a gene called MECP2, which is located on the X chromosome. Patients lose coordination, mobility, and their ability to ...
NEW YORK--(BUSINESS WIRE)--Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, ...
TORONTO, Nov. 01, 2022 (GLOBE NEWSWIRE) -- PharmaTher Holdings Ltd. (the “Company” or “PharmaTher”) (OTCQB: PHRRF) (CSE: PHRM), a specialty pharmaceutical company focused on developing and ...
MONEY AND AWARENESS. CONCORD HIGH SCHOOL STUDENT IZZY BOOM IS BUSY WITH HER FRIENDS EMMA AND SAMMY. OH YEAH, I THINK THIS ONE’S MY FAVORITE DECORATING DUCKS. OH, THAT’S A CUTE ONE. TWO, TWO DUCK. BUT ...
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