GlobalData on MSN1mon
Italian charity seeks EMA approval for rescued gene therapy
Italian research charity Telethon Foundation is one step closer to getting a gene therapy to treat rare disease ...
Hosted on MSN4mon
Stem cells can tailor their role in gene therapy based on the underlying disease, study suggests
Researchers analyzed 53 patients treated with lentiviral HSC-GT for metachromatic leukodystrophy, Wiskott-Aldrich syndrome, or β-thalassemia. Patients were followed for up to eight years ...
Forbes Advocate8d
Local young warrior sparks awareness for Rare Disease Day
On Friday, 14 March 14, IDFA CEO Carolyn Dews and Emma Baxter, mother of local young warrior Jack Baxter, will visit the ...
Nature5y
The immune sting of WASp
Despite its name, the Wiskott-Aldrich syndrome protein (WASp) is not only implicated in Wiskott-Aldrich syndrome, a rare genetic disease characterized by low platelet count, eczema and recurrent ...