Ultragenyx Pharmaceutical Inc

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Many hands make light work: why the new Bespoke Gene Therapy Consortium is a godsend for ambitious biopharma teams

21 December 2021 | By Christian K Schneider (Biopharma Excellence), Christian K Schneider (PharmaLex)

In late October, we learned that an exciting and ambitious new entity – the Bespoke Gene Therapy Consortium (BGTC) – had come into being in the US, designed to encourage the delivery of more gene therapies for rare diseases. The consortium is an interesting construct, headed and funded by the…

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FDA joins Bespoke Gene Therapy Consortium (BGTC) for rare diseases

28 October 2021 | By Anna Begley (European Pharmaceutical Review)

The consortium also consists of 10 pharmaceutical companies and five organisations to accelerate gene therapies to treat rare diseases.

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FDA approves first therapy for rare inherited form of rickets, x-linked hypophosphatemia

20 April 2018 | By Dr Zara Kassam (European Pharmaceutical Review)

The FDA has approved Crysvita, the first drug approved to treat adults and children ages 1 year and older with x-linked hypophosphatemia, a rare, inherited form of rickets...