Yahoo

What Is Angelman Syndrome? Everything to Know About the Rare Genetic Disorder Colin Farrell’s Son James Has

For the first time, Colin Farrell has opened up his home and talked in-depth about life with his son James, who has Angelman syndrome. James was just a toddler when he was diagnosed with the rare ...
Seeking Alpha

Ionis treatment for Angelman syndrome receives orphan drug and rare pediatric disease designations from U.S. FDA

CARLSBAD, Calif., June 13, 2022 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (IONS), the leader in RNA-targeted therapeutics, announced today that the U.S. Food and Drug Administration (FDA) has ...
Morningstar

Ultragenyx Completes Enrollment of Phase 3 Aspire Study Evaluating GTX-102 for the Treatment of Angelman Syndrome

Company expects to complete Phase 3 Aspire study in the second half of 2026 Aurora study of GTX-102 in additional ages and genotypes on track to initiate in the second half of 2025 NOVATO, Calif., ...
Morningstar

MavriX Bio Receives FDA Fast Track Designation for MVX-220 for Treatment of Angelman Syndrome

MIDDLETON, Mass., Sept. 22, 2025 /PRNewswire/ -- MavriX Bio, a clinical-stage biotechnology company focused on the development of transformative genetic therapies for Angelman syndrome (AS), today ...
STAT

Ultragenyx reports early success with Angelman syndrome drug, backing up 2022 acquisition

Ultragenyx has found early success with its experimental treatment for the rare disease Angelman syndrome, supporting the company’s decision to acquire its development partner two years ago.
Business Wire

Ovid Therapeutics and NeuroPointDX Announce Collaboration to Identify Biomarkers for Angelman Syndrome

Angelman syndrome is a rare genetic disorder that is characterized by a variety of signs and symptoms, including delayed development, intellectual disability, severe speech impairment, problems with ...
News Medical

Small molecule discovery offers hope for treatment of Angelman syndrome

Angelman syndrome is a rare genetic disorder caused by mutations in the maternally-inherited UBE3A gene and characterized by poor muscle control, limited speech, epilepsy, and intellectual ...
Nasdaq

Ultragenyx: 1st patient dosed in Aspire studyof GTX-102 for Angelman treatment

Ultragenyx (RARE) Pharmaceutical announced that the first patient has been dosed in the pivotal Phase 3 Aspire study, NCT06617429, evaluating the efficacy and safety of GTX-102, its investigational ...
Hosted on MSN

Ultragenyx doses first subject in study of GTX-102 for angelman syndrome

Ultragenyx Pharmaceutical has dosed the first participant in Aurora, a study evaluating GTX-102 (apazunersen) for angelman syndrome (AS). The rare, neurogenetic condition results from the loss of ...
Business Insider

Ultragenyx Announces Successful End-of-Phase 2 Meeting with FDA for GTX-102 Angelman Syndrome Program

Alignment with FDA on Phase 3 study primary endpoint of Bayley-4 cognition and key secondary endpoint of Multi-Domain Responder Index (MDRI) Phase 3 study on track to initiate by the end of this year ...
PR Newswire

Angelman Syndrome Market to Witness Significant Growth Through 2034, Driven by Novel Therapeutic Advancements | DelveInsight

According to DelveInsight's estimates, the Angelman syndrome market size in the 7MM is expected to show positive growth during the forecast period (2025–2034), primarily driven by the increasing ...