Ultragenyx’s Angelman therapy fails Phase 3 trial in setback for
In a major setback to the search for a treatment for the rare neurodevelopmental disease known as Angelman syndrome, Ultragenyx said a Phase 3 trial of its experimental therapy failed. The company's antisense oligonucleotide therapy ...
Ultragenyx's experimental therapy for Angelman syndrome, a rare neurodevelopmental disease, has failed a Phase 3 trial. The therapy, GTX-102, an antisense oligonucleotide, showed no benefit compared to a sham treatment.
The failure is a significant setback for the search for a treatment for Angelman syndrome, which causes severe intellectual disabilities and developmental delays. According to STAT News, the therapy had shown powerful results in early trials, raising hopes for families affected by the condition.
The news is also a blow to Ultragenyx's business, as the company had been relying on the Angelman drug as a path to profitability, despite having multiple approved medicines for ultra-rare diseases.
Brief written by urgent.news from Endpoints News, STAT News — 2 reports on this story. Machine-written — may contain errors; check the original before relying on it.
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