There is still no cure for DMD so far and the standard of care is principally limited to symptom relief through glucocorticoids treatments. Current therapeutic strategies could be divided into two ...

cNeuromuscular and Neurogenetic Disorders of Childhood Section, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD, USA dDubowitz Neuromuscular Centre ...

For our high-throughput screening campaign, we utilized an NSC34 motor neuron cell line expressing an SMN2 minigene reporter. A pair of SMN2 matched reporter constructs were designed such that one ...

Advancements in gene therapy for Duchenne muscular dystrophy (DMD) have improved the outlook for some patients, but further ...

Abstract: The previous five years have shown a rapidly growing interest in digital micromirror device (DMD) as a spatial light modulator for complex photonics applications, primarily due to their high ...

M Khorshed Anowar, DMD and Head of Retail and SME Banking of EBL, and Md Sohrawardi, Director of ZSRM Group, signed an agreement to this effect at the EBL head office in Dhaka. Ahmed Shaheen, ...

My grandson William has Duchenne muscular dystrophy (DMD), a condition that comes with a set of expectations about how it will progress. Doctors provided guidelines about what we might expect—gradual ...

At the 28th American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, Scribe showcased the potential of its X-Editor (XE) genome editing technology to target the underlying genetic causes of ...

The one-time gene therapy Elevidys (delandistrogene moxeparvovec-rokl) was associated with stabilizations in motor function for boys with Duchenne muscular dystrophy (DMD) who were treated when they ...

NEW ORLEANS – EdiGene on Saturday revealed early clinical trial results from the first two patients treated with LE051, an RNA-editing drug it is developing for Duchenne muscular dystrophy (DMD). The ...

The multi-cohort ENDEAVOR study is aimed at evaluating the therapy’s safety and protein expression in male subjects with DMD. Across seven cohorts, the trial enrolled 55 subjects and dosed ...