Despite mixed results using gene therapies to treat Duchenne muscular dystrophy, drug developers are pushing ahead with the ...

Muscular dystrophy is caused by a lack of certain proteins that are needed for muscle structure and function. Specifically, many of the types of MD are caused by a lack of a protein called dystrophin, ...

It offers EXONDYS 51 injection to treat duchenne muscular dystrophy (duchenne) in patients with confirmed mutation of the dystrophin gene that is amenable to exon 51 skipping; VYONDYS 53 for the ...

Children's Mercy Kansas City has achieved a significant advancement toward the treatment of rare genetic diseases through the ...

Analysts at William Blair boosted their FY2024 earnings per share estimates for shares of Sarepta Therapeutics in a research ...

The US Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation to NS-051/NCNP-04, which is ...

Dyne Therapeutics is a clinical stage biotech concern with just over $700 million of cash on its balance sheet. Learn more ...

The Shanghai-based firm will test the gene therapy candidate in patients between 4 and 9 years old with Duchenne muscular dystrophy.

该研究也指出，由于DMD突变类型多样且分布广泛，单一ASO药物难以覆盖所有患者。尤其是针对深层内含子突变的修复，现有方法的效果仍然有限。此外，基因治疗的研发成本高昂，临床转化周期长，给患者的普及使用带来显著障碍。

The one-dose gene therapy – much like rivals from Pfizer and Solid Biosciences – codes for a shortened form of dystrophin that is deficient in patients with the X-linked muscle-wasting ...

According to Dr Ann Agnes Mathew, Consultant Pediatric Neurologist at Bangalore Baptist Hospital, DMD is life-threatening and ...

In this review of RNA therapies, we paint an overview of what’s going on in a field that is addressing a number of diseases.