Amerikansk kraftsamling för nya genterapier mot sällsynta sjukdomar

USA:s hälsomyndigheter har tillsammans med tio läkemedelsbolag och fem intresseorganisationer gått samman för att snabba upp utvecklingen av genterapier för sällsynta sjukdomar.

By Anders Göransson

November 1, 2021 10:16 AM

I USA lider ungefär 30 miljoner människor av någon av de omkring 7 000 sällsynta sjukdomar man känner till. Men godkända genterapier finns idag för endast två av dem.

Det nyligen lanserade Bespoke Gene Therapy Consortium, BGTC, (ung. Konsortiet för skräddarsydd genterapi) vill ändra på det genom att förbättra och effektivisera utvecklingsprocessen för genterapier.

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