About DT-216P2 DT-216P2 is an improved formulation of a GeneTAC ® small molecule designed to specifically target the GAA repeat expansion mutation that is the underlying cause of FA and restore the ...

Researchers have developed a gene editing strategy that can repair the genetic problems that lead to Huntington's disease and Friedreich's ataxia. | Genetics And Genomics ...

Figure 4: Correlation between the size of expanded SCA10 ATTCT repeat and the age of onset. Figure 5: Sca10 is widely expressed in brain regions that are anatomical substrates for ataxia and epilepsy.

After getting a green light from the European Commission, Biogen’s Skyclarys is the first approved medicine for the inherited neurological disease Friedreich’s ataxia (FA) in the EU.

Biotechnology is a broad discipline in which biological processes, organisms, cells or cellular components are exploited to develop new technologies. New tools and products developed by ...

Dostarlimab with platinum-based chemotherapy for treating primary advanced or recurrent endometrial cancer with high microsatellite instability or mismatch repair deficiency TA1064 22 May 2025 22 May ...

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Larimar is taking part in the pilot with its nomlabofusp protein replacement therapy for Friedreich’s ataxia (FA), a devastating and progressive disease caused by mutations that lead to low ...

June 3, 2025 — A new study challenges a decades-old assumption in neuroscience by showing that the brain uses distinct transmission sites -- not a shared site -- to achieve different types of ...

Even loyalty programs (buy 10 slices of pizza, get the 11th free) and coupons (10 percent off your next visit) are ways to drive repeat business and recurring revenue.” People are inundated with ...

May 29, 2025 — Scientists have documented the way a single gene in the bacterium that causes bubonic plague, Yersinia pestis, allowed it to survive hundreds of ... New AI Tool Reveals Single ...