The Duchenne muscular dystrophy (DMD) landscape across the seven major markets (7MM) - the US, France, Germany, Italy, Spain, ...

During a live event, Alexander I. Spira, MD, PhD, and participants NGS testing practices and the rationale behind their ...

Its expansion is attributed to the treatment of ambulatory DMD patients. The Duchenne muscular dystrophy (DMD) market across ...

This valuable work presents how PRDM16 plays a critical role during colloid plexus development, through regulating BMP signaling. Solid evidence supports the context-dependent gene regulatory ...

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

Mammoth Biosciences researchers have developed NanoCas, an ultracompact CRISPR nuclease, demonstrating its ability to perform ...

Ridgefield, Conn., U.S., and Ingelheim, Germany Zongertinib would be the first orally administered, targeted therapy for previously treated patients with HER2 (ERBB2)-mutant advanced non-small cell ...

In 1958, Francis Crick's sequence hypothesis finally provided an answer to the question: what is a gene? Why is this definition now considered overly simplistic? Roberts and Sharp also noted that ...

Dec. 10, 2024 — In a single IV injection, a gene therapy targeting cBIN1 can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the ...

This important study examines the role of pericytes in patterning the zebrafish blood-brain barrier (BBB) and controlling its permeability. Using pdgfrb mutant zebrafish models lacking brain pericytes ...

Gene regulation refers to the mechanisms that act to induce or repress the expression of a gene. These include structural and chemical changes to the genetic material, binding of proteins to ...