“Beyond UniQure, the pipeline includes highly promising approaches that will ideally move the goal from merely managing ...

Until recently, even the most advanced gene therapies could only be given after a child was born—often racing against time to prevent irreversible damage. In the first part of this series, we explored ...

A new study has brought scientists one step further in the direction of developing a cure for a brutal group of rare brain ...

As newborn screening and rapid DNA sequencing become routine, we are poised to catch and treat inherited diseases at their earliest stages. Today, we can intervene in the first days or weeks of life.

A CRISPR-Cas9-based therapy targeting the gene encoding transthyretin greatly reduced transthyretin levels in patients with ...

Following up on previous, dimly received issuances, a new set of ideas published by the FDA to streamline regulatory pathways ...

Alison Clare has received funding from the Wellcome Trust and the Medical Research Council. Her position is currently funded by National Institute for Health and Care Research (NIHR) Biomedical ...

DMD gene therapy faces challenges in safety, efficacy, and long-term outcomes, despite FDA approval of delandistrogene moxeparvovec. Future strategies focus on novel AAV capsids, dual/triple vector ...

Myrtelle Inc. ("Myrtelle" or the "Company"), a gene therapy company pioneering transformative treatments for ...