Italian non-profit organization Fondazione Telethon today announced the European Commission’s decision of January 9, to grant ...
What Is Waskyra, and Why Does It Matter? Waskyra (etuvetidigene autotemcel) is a new gene therapy approved to treat Wiskott-Aldrich syndrome (WAS), a rare inherited condition that affects the immune ...
Fondazione Telethon announces the positive opinion issued by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) recommending marketing authorisation in ...
The U.S. Food and Drug Administration has approved Waskyra (etuvetidigene autotemcel) as the first cell-based gene therapy for the treatment of Wiskott-Aldrich syndrome (WAS). RCCS San Raffaele ...
With the decision by the U.S. Food and Drug Administration and the European Commission to grant market authorization for ...
2025 would also go down in history as the year when gene therapies began to go mainstream, with multiple of them being approved, writes Satyen K. Bordoloi My first memory of cancer is the younger ...
The MarketWatch News Department was not involved in the creation of this content. -- WAS affects almost exclusively males, with an estimated incidence of 1 in 250,000 live male births. -- The therapy ...
The FDA on Tuesday approved etuvetidigene autotemcel (Waskyra) as the first cell-based gene therapy for patients 6 months and older with Wiskott-Aldrich syndrome, a rare primary immunodeficiency. The ...
Robin Alderman faces an agonizing reality: Gene therapy might cure her son Camden’s rare, inherited immune deficiency. But it’s not available to him. In 2022, London-based Orchard Therapeutics stopped ...
Fondazione Telethon announced today that the U.S. Food and Drug Administration (FDA) has approved the Biologics License Application (BLA) for Waskyra, an ex vivo gene therapy for patients with Wiskott ...
The US FDA has approved the first ever cell-based gene therapy to treat Wiskott-Aldrich syndrome (WAS). The gene therapy, Waskyra (etuvetidigene), can be used to treat adults with WAS who have a ...
Gene therapy may cure rare diseases. But drugmakers have few incentives, leaving families desperate.
Robin Alderman faces an agonizing reality: Gene therapy might cure her son Camden’s rare, inherited immune deficiency. But it’s not available to him. In 2022, London-based Orchard Therapeutics stopped ...
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