Ultragenyx Completes Enrollment of Phase 3 Aspire Study Evaluating GTX-102 for the Treatment of Angelman Syndrome
Ultragenyx announces the Phase 3 ASPIRE study for apazunersen (GTX-102) is fully enrolled with 129 participants, reaching this milestone in just seven months.
Inside INSYNC-AS 2025: Progress, Partnership, and Possibilities for Angelman Syndrome
The 5th Annual INSYNC-AS Meeting brought together top researchers to tackle brain-targeted drug delivery, sharing breakthroughs in blood-brain barrier crossing for Angelman syndrome therapies.
Oak Hill Bio Announces Publication of Rugonersen Phase 1 Study Results in Nature Medicine
Oak Hill Bio’s Phase 1 TANGELO study results, published in Nature Medicine, show improvements in brain activity and development for children with Angelman syndrome on rugonersen.
Ultragenyx Receives Breakthrough Therapy Designation for GTX-102 in Angelman Syndrome
Ultragenyx’s GTX-102 (apazunersen) has received FDA Breakthrough Therapy Designation for Angelman syndrome, marking a key milestone in its path toward potential approval.
Investing in the Future of Angelman Syndrome Research: FAST Partners with ASGCT to Support Early-Career Scientists
FAST partners with ASGCT to fund up to three $100,000 Career Development Awards, recruiting early-career scientists to advance neuron-targeted gene and cell therapies for Angelman syndrome.”
First patient dosed in pivotal Phase 3 REVEAL clinical study of ION582 in Angelman syndrome
Ionis Pharmaceuticals has dosed the first participant in its global Phase 3 REVEAL trial, evaluating the safety and efficacy of ION582 as a potential treatment for Angelman syndrome.
FAST Announces Funding For First Innovative Seed Grant
FAST has awarded its first Innovative Seed Grant to Drs. Rachel Cunningham and Jennifer Adair to develop a safer, more accessible hematopoietic stem cell therapy approach for Angelman syndrome that avoids risky chemotherapy-based conditioning.
Leading Neurology and Rare Disease Expert Dr. Nick France Joins FAST Board
FAST welcomes Dr. Nick France, a rare disease drug development leader from Alexion, AstraZeneca Rare Disease, to its Board of Directors, bringing two decades of biopharmaceutical and gene therapy expertise.
CAN Spotlight: Kettlebells for Kayleigh Fundraiser in Germany
Crossfitters from all around the state joined the Gainey family at their 2nd annual Kettlebells for Kayleigh, a fundraising event to benefit Angelman syndrome in Kaiserslautern, Germany. This event doubled in success from their first year and gained more supporters from around the area and with local media. Over $7,000 USD was raised to benefit […]
CAN Spotlight: Skating Angels Charity Ice Hockey Tournament
Danielle and Jeff hosted another successful Skating Angels event to benefit FAST, raising over $18,000 for the Angelman syndrome community through adult and mini 3v3 hockey tournaments.