– The Company today reported positive initial results from the ongoing FREEDOM-DM1 trial in patients with DM1; mean splicing correction of 12.3% and 29.1% in 5 and 10 mg/kg cohorts, respectively ...
BOSTON, February 24, 2025--(BUSINESS WIRE)--PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of ...
– The Company today reported positive initial results from the ongoing FREEDOM-DM1 trial in patients with DM1; mean splicing correction of 12.3% and 29.1% in 5 and 10 mg/kg cohorts, respectively – – ...
Department of Biochemistry, Duke University School of Medicine, Durham, North Carolina 27705, United States ...
Here, we report the generation and characterization of a novel Huntington’s disease (HD) mouse model BAC226Q by using a bacterial artificial chromosome (BAC) system, expressing full-length human HTT ...
Motor neuron diseases (MND) refer to a wide and heterogeneous expanding group of neurodegenerative disorders involving the upper and lower motor neurons, which are characterized by rapidly progressive ...
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