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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 ...

Ultragenyx Announces First Patient Dosed in Aurora Study Evaluating GTX-102 in Additional Angelman Syndrome Genotypes and Age Groups

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that the first patient has been dosed in the Aurora study (NCT07157254) evaluating the efficacy and safety of GTX-102 (apazunersen), an ...

Abby McCloskey: New IVF policies bring hope — and moral questions

Scientists are on the brink of even preventing some diseases — not just treating them — at the sperm, egg and early embryo ...
Business Wire

Ionis receives U.S. FDA Breakthrough Therapy designation for ION582 in Angelman syndrome

CARLSBAD, Calif.--(BUSINESS WIRE)--Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to ION582 ...
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 ...
Benzinga.com

Ionis Pharmaceuticals To Expedite Development Of Rare Neurological Disease Candidate With FDA Breakthrough Status

On Tuesday, the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation to Ionis Pharmaceuticals Inc.’s (NASDAQ:IONS) ION582 for Angelman syndrome (AS), a rare neurological ...