Latest Conference Coverage

The data warrant further evaluation of ALLO-316 in patients with confirmed CD70+ tumors.

The professor for human genetics and neurology at the University of Miami Miller School of Medicine discussed the rapidly changing landscape of diagnosing and treating genetic disorders.

The professor for human genetics and neurology at the University of Miami Miller School of Medicine discussed past, current, and future approaches to diagnosing and treating neuromuscular diseases.

The professor for human genetics and neurology at the University of Miami Miller School of Medicine discussed the trend towards gene-targeted approaches in the neuromuscular disease space.

The assistant professor of medicine at Vanderbilt University Medical Center discussed the work that has been done in the field in the past 10 years and work that still remains to be done.

The executive vice president and chief research officer of MDA discussed the barriers to FDA approval currently facing those developing treatments for ultra-rare diseases.

The director of the center for gene therapy at Nationwide Children's Hospital discussed the prospect of gene therapy approvals in Duchenne muscular dystrophy and the associated challenges that remain to be addressed.

Peter Marks, MD, PhD, director, Center for Biologics Evaluation and Research, FDA, discussed his keynote address at the 2023 MDA Conference. c

The locum consultant of pediatric BMT and leukemia at Royal Manchester Children’s Hospital discussed the center’s experience in delivering the approved gene therapy to children with MLD.

The co-CEO of BrainStorm Cell Therapeutics spoke about the current standards of care in ALS and the potential of NurOwn to address unmet needs.

The associate professor of neurology at Columbia University spoke about the importance of identifying patients with ALS-associated gene mutations as early as possible.

The Lillehei professor in stem cell and regenerative cardiovascular medicine at the University of Minnesota spoke about preclinical research with an iPS cell-derived product.

The associate professor of neurology at Columbia University spoke about the current and future treatment landscape in ALS.

The Lillehei professor in stem cell and regenerative cardiovascular medicine at the University of Minnesota spoke about the session she chaired at the 2023 MDA Clinical and Scientific Conference.

Krystal Biotech's investigational topical gene therapy has shown promise in DEB. It is designed to deliver COL7A1 and restore C7 protein in patients with the rare disorder.

The co-CEO of BrainStorm Cell Therapeutics spoke on the supporting data for the investigational ALS cell therapy that will be discussed at the meeting, and expectations for it.

The nurse practitioner from Washington University in St. Louis discussed the practical considerations in gene therapy track she chaired at the 2023 MDA Conference.

The Delphi panel considerations were informed by safety data from 3 clinical trials of the therapy, and included vomiting, myocarditis, acute liver injury, and immune-mediated myositis.

The co-founder and chief executive officer of MyoGene Bio spoke about the company's gene editing therapy for Duchenne muscular dystrophy at MDA’s 2023 conference.

The Amplo Biotechnology gene therapy showed positive findings in mouse models, suggestive of the therapy’s development for use in a clinical setting. The AAV therapy delivers DOK7 gene, which has shown benefit in neuromuscular disorders.

Data from the HOPE-2-OLE were presented in a late-breaking session at the 2023 MDA Conference.

All children included in the analysis achieved swallowing, oral nutrition, and airway protection outcomes.

The chief medical officer of QurAlis discussed the antisense oligonucleotide therapy QRL-201.

Individual items on RULM revealed meaningful improvements in participants from the SHINE and CHERISH studies.

The professor of neurology and pediatrics at University of Rochester Medical Center spoke about her session at MDA’s 2023 conference.

New data from a single-site, first-in-human study were presented at the 2023 MDA Conference.

The director of the Center for Biologics Evaluation and Research at the FDA discussed his keynote speech at the 2023 MDA Conference.

Data from the phase 4 RESPOND trial were presented at the 2023 MDA Conference.