A 12-year-old boy with a severe form of muscular dystrophy has said it is "cruel" that a new drug is not being made available ...

研究人员首次发现，人类细胞自带一套精密的"应急修复系统"，当检测到关键基因故障时，会自动激活"替补基因"展开修复。更令人振奋的是，这项机制可被人工干预精确调控，为DMD乃至6000多种单基因遗传病带来 ...

Medically reviewed by Smita Patel, DO Duchenne muscular dystrophy (DMD) is a rare neurological condition that causes severe muscle weakness and intellectual disability. DMD is an inherited (passed ...

Duchenne muscular dystrophy (DMD) is a rare neurological condition that causes severe muscle weakness and intellectual disability. DMD is an inherited (passed down) disorder. The condition is ...

转录适应上调杜氏肌营养不良症（DMD）患者的UTRN，这得到了几条证据的支持，包括使用剪接开关反义寡核苷酸诱导DMD基因框外外显子的跳跃。

The company raised $200 million after early findings hinted its therapy could be more potent than others like it, including ...

Solid said that the first patients to receive its Duchenne gene therapy all produced high levels of the target protein, an ...

Duchenne muscular dystrophy (DMD) is a rare hereditary disease that is associated with progressive muscle wasting. The ...

Day 90 biopsy data reported from first 3 participants dosed in Phase 1/2 INSPIRE DUCHENNE trial —— Average microdystrophin ...

Families and doctors spoke out against the cuts at a roundtable at the UMass Chan Medical School in Worcester, Massachusetts.

Satellos Bioscience Inc. (TSX: MSCL, OTCQB: MSCLF) (“Satellos” or the “Company”), a public biotech company developing new ...