The co-parents came together to address an important issue with their girls Leah Messer and Corey Simms are showing a united ...

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

Less than half of patients had undergone bone monitoring, suggesting a need for better clinical guidance and management of ...

Understanding the behavior of tiny droplets in our cells could aid the search for new treatments. A team of Leiden ...

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。

Data from DELIVER and ACHIEVE Clinicals Trial to be Presented -- Presentation on ACHIEVE Trial in DM1 and Company Symposium to Feature Data on ...

About Evrysdi Evrysdi is an SMN2-splicing modifier designed to increase functional SMN protein production. With the introduction of a new tablet formulation, Evrysdi now offers an alternative ...

Results show improved functional outcomes for people with Duchenne muscular dystrophy who are ambulatory. Elevidys, which is ...

On March 18 th Jerel A. Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec will be giving an oral presentation in the first session dedicated to OPMD at the 2025 Muscular ...