Fierce Pharma

With pivotal trial underway, Ionis ramps up Angelman syndrome awareness with Lifetime TV segment

As it kicks off a pivotal phase 3 trial of its investigational treatment for Angelman syndrome (AS), Ionis Pharmaceuticals is ...

Encoded Therapeutics Presents Positive Interim Efficacy Data from Initial Dose Levels of Phase 1/2 Trials Evaluating ETX101 Gene Therapy in Dravet Syndrome

To date, ETX101 has shown a favorable safety profile and has been well-tolerated across all four dose levels with no treatment-related serious adverse events (n=19). The most common treatment-related ...

Marathon runner covers 1100km across NSW to raise awareness for Angelman Syndrome and Huxley’s Heroes

Marathon runner covers 1100km across NSW to raise awareness for Angelman Syndrome and Huxley’s Heroes ...
WCPO Cincinnati

Cincinnati man goes on cross-country mission to find a cure for rare syndrome

Cincinnati native Charlie Nuck has a 6-year-old niece named Briar. She has a rare, neurogenetic disorder called Angelman syndrome. It affects about one in 15,000 people and causes developmental delays ...
Yahoo Finance

Ultragenyx’s GTX-102 Receives FDA Breakthrough Therapy Designation for Angelman Syndrome

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) is one of the most promising stocks according to Wall Street analysts. On June 27, Ultragenyx Pharmaceutical announced that the US FDA granted Breakthrough ...
Yahoo Finance

Ultragenyx Receives Breakthrough Therapy Designation for GTX-102 in Angelman Syndrome

NOVATO, Calif., June 27, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), today announced that it has received Breakthrough Therapy Designation from the U.S. Food and Drug ...
Concord Monitor

Bowling for a cause: Angelman Syndrome Fundraiser coming to Boutwell’s

3-year-old Bellamy Bresaw of Bow was diagnosed with Angelman Syndrome at nine months old. Credit: Delores Flanders / Courtesy Delores Flanders and her family are hosting a fundraiser at Boutwell’s ...
ABC12

Montrose family raising money for 6-year-old boy with Angelman Syndrome

MONTROSE, Mich. (WJRT) - A Mid-Michigan family is raising money to help their 6-year-old boy with a rare genetic disorder. After spending a few months looking for action figures and fun items, Kristin ...
Seeking Alpha

Ultragenyx posts trial data backing Phase 3 design for Angelman syndrome therapy

Ultragenyx Pharmaceutical (NASDAQ:RARE) announced Saturday that GTX-102, its candidate for the rare neuro-genetic disorder Angelman syndrome, improved patient outcomes across multiple domains in a ...
Nasdaq

Ionis Announces Pivotal Phase 3 Trial Design for ION582 in Angelman Syndrome

Positive End of Phase 2 discussion with FDA, including alignment on Phase 3 design Bayley-4 expressive communication selected as Phase 3 study primary endpoint Initiation of ION582 Phase 3 study ...
Reporter-Herald

Loveland man with Angelman syndrome to compete in Chicago Marathon

PJ Snyder, a 38-year-old man with Angelman syndrome, is nonverbal, can walk only with assistance, and has a seizure disorder. He will also compete in the Chicago Marathon next month. In fact, through ...