In an article published in Molecular Cell, scientists have discovered how the three-dimensional organization of DNA in our ...
As many as 40% of Americans are obese, putting them at an increased risk for high blood pressure, diabetes, stroke, heart disease and certain cancers, according to the CDC.
There is an unmet need for therapies targeting Duchenne muscular dystrophy (DMD)–related heart disease, and phase 2 results ...
The lab of The Wistar Institute's Jessie Villanueva, Ph.D., has identified a new strategy for attacking treatment-resistant ...
While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several ...
Gene therapies have set a high bar for outcomes in pharma by delivering curative treatments for difficult diseases in a one-and-done punch. But for Duchenne muscular dystrophy, the results of ...
NEW YORK – Belief BioMed announced Wednesday that the US Food and Drug Administration has cleared its investigational new drug application enabling it to begin testing its gene therapy candidate for ...
Certain types, including Duchenne muscular dystrophy, can lead to problems with the heart and ... It often makes the shoulders slope forward and causes difficulty raising the arms. A gene mutation is ...
While DMD is currently incurable, hope lies in emerging treatments like ELEVIDYS (Delandistrogene Moxeparvovec Rokl), a gene therapy designed to help the muscles produce dystrophin. However ...
This type of shuffling has been challenging to detect due to the high similarity between DNA sequences. The study also identifies specific regions of DNA that remain largely unchanged, likely serving ...