Charlie Nuck of Cincinnati has a 6-year-old niece named Briar. She has a rare, neurogenetic disorder called Angelman syndrome ...

An Ohio uncle is on a cross-country mission, riding through Northeast Ohio this weekend to find a cure for a rare syndrome.

On June 27, Ultragenyx Pharmaceutical announced that the US FDA granted Breakthrough Therapy Designation for GTX-102 ...

NOVATO, Calif., June 27, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), today announced that it has ...

Prader-Willi syndrome is a study in contrast — children who once couldn’t eat later battle an urge to never stop. The ...

Phase 3 Aspire study enrollment on track to complete in 2025 Aurora study to evaluate GTX-102 in other Angelman syndrome genotypes and ages expected to initiate later this year NOVATO, Calif., June 27 ...

In a conversation with People magazine in 2024, Colin Farrell opened up for the first time about his son's rare condition: Angelman syndrome. Just before his son James’ 21st birthday, the actor ...

A new review sheds light on the complex molecular mechanisms behind Angelman syndrome (AS), a rare neurogenetic disorder, and explores how cutting-edge gene-targeting therapies may offer more ...

MIDDLETON, Mass., May 12, 2025 /PRNewswire/ -- MavriX Bio, a clinical-stage biotechnology company focused on the development of transformative genetic therapies for Angelman syndrome (AS), today ...

MavriX Bio, a clinical-stage biotechnology company focused on the development of transformative genetic therapies for Angelman syndrome (AS), today announced that the U.S. Food and Drug ...

MavriX Bio Announces FDA Clearance of IND Application to Initiate First-in-Human Study of Gene Therapy for Angelman Syndrome ...

MavriX Bio Announces FDA Clearance of IND Application to Initiate First-in-Human Study of Gene Therapy for Angelman Syndrome By MavriX Bio May 12, 2025 Updated May 12, 2025 Logo for Mavrix Bio ...