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This therapeutic approach is in an advanced stage of development for another immunodeficiency, Wiskott-Aldrich syndrome. Other diseases on which the gene therapy developed by Telethon researchers ...

This therapeutic approach is in an advanced stage of development for another immunodeficiency, Wiskott-Aldrich syndrome. Other diseases on which the gene therapy developed by Telethon researchers ...

Can-Fite and Fondazione Telethon have signed a collaboration agreement for the clinical development of Piclidenoson for the treatment of Lowe Syndrome, a high medical need with no drug available.

The BP1-BP5 segment includes the Prader-Willi Syndrome (PWS) and the Angelman Syndrome (AS) imprinted regions as shown in Figure 2. These two syndromes differ in phenotype and are caused by the ...

Aldrich Potgieter – who’s a first-timer in the field this week at THE PLAYERS Championship 2025 – has an equipment setup that’s built for control, not speed, but he still currently ranks ...

for the gene therapy - etuvetidigene autotemcel – as a treatment of patients with Wiskott-Aldrich syndrome (WAS), a rare genetic disease of the immune system, to the US Food and Drug Administration ...