14 Nov – Fred Cheng recently celebrated turning 41 by revealing that his focus this year has been on son Asher.

Phase 1/2 data show improvements across all domains and confirm that Phase 3 Aspire study is amply powered to establish ...

Ultragenyx Pharmaceutical (RARE) shows promising results for GTX-102 in Angelman syndrome Phase 1/2 trial, supporting a Phase ...

Ultragenyx Pharmaceutical Inc. (RARE) announced Phase 1/2 data in support of the Phase 3 Aspire study for GTX-102, its ...

She’s made her name counting dollars and cents for tens of thousands of Australians, helping to manage their budgets and ...

Ionis has committed to a phase 3 programme for its antisense therapy for rare genetic disorder Angelman syndrome, just weeks after Biogen decided against exercising an option to license the drug.

The context: The board said it decided to deploy cash of up to $50 million to buy back shares due to its “sustainable and ...

A four-year-old girl with DUP15q syndrome, who doctors said would never walk, has defied expectations to take her first steps.

Joseph Schwartz has given his Buy rating due to a combination of factors surrounding Ultragenyx Pharmaceutical’s GTX-102 program for Angelman syndrome. The updated data from the Phase 1/2 trial, ...

It primarily develops TSHA-120 for the treatment of giant axonal neuropathy; TSHA-102 for the treatment of Rett syndrome; TSHA-121 for the treatment of CLN7 disease; TSHA-118 for the treatment of CLN1 ...