A recent study highlights de novo facioscapulohumeral muscular dystrophy as a severe subtype linked to D4Z4 repeat contractions. This discovery enhances understanding of the genetic mechanisms underlying this condition.

A response has been published addressing concerns raised in a letter to the editor about mortality associated with facioscapulohumeral muscular dystrophy (FSHD). This systematic literature review highlights the complexities of mortality in FSHD, contributing to the ongoing discourse in the scientific community.

Epicrispr secures funding to support clinical development of EPI-321, a gene therapy targeting muscular dystrophy treatment Ultimately, our vision is to establish programmable epigenetic medicines as an entirely new therapeutic modality, with the potential to treat diseases that have historically been beyond the reach of conventional approaches.” · Related:Poolbeg CEO delves inside the UK’s record $2.84bn fundraising quarter ... The LSX USA Congress gathers leaders from the US biotech, medtech, and healthtech sectors to network, showcase innovations, and explore partnership opportunities. Related:Vaderis secures $152m to advance rare disease treatment · EPI-321 is designed to be delivered intravenously via a single adeno-associated virus (AAV) vector. The drug aims to durably suppress pathological DUX4 expression without permanently altering the underlying DNA sequence. This Series C marks a shift in the company's fundraising approach compared with earlier rounds, according to Salzman. Facioscapulohumeral muscular dystrophy (FSHD) is one of the most common forms of muscular dystrophy, affecting hundreds of thousands of people worldwide. There are currently no approved disease-modifying therapies. Salzman shared that the disease is a perfect match for the company's approach.

Epicrispr Biotechnologies and Infinimmune have secured a combined $165 million in venture capital funding. Epicrispr aims to develop an epigenetic treatment for facioscapulohumeral muscular dystrophy, while Infinimmune will focus on advancing antibodies for atopic dermatitis.

Epicrispr secures $90 million in funding to advance its epigenetic editing drug for facioscapulohumeral muscular dystrophy. The company has completed enrollment for an early-stage study, positioning itself in a competitive landscape with multiple drugmakers targeting this rare muscle disease.