Latest Conference Coverage

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 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.

The associate professor of clinical pediatrics at Cincinnati Children's Hospital Medical Center discussed Sangamo Therapeutics’ gene therapy, ST-920, and the phase 2 STAAR clinical trial.

Catch up with some of CGTLive’s top news articles from the annual WORLDSymposium.

The associate professor of clinical pediatrics at Cincinnati Children's Hospital Medical Center discussed the limitations of the current standard of care for Fabry disease.

Matthew B. Harms, MD, associate professor of neurology at Columbia University, and medical consultant and care center director at the MDA, discussed the session he will be chairing at the conference.

Catch up with some of CGTLive’s top conversations from the annual ASTCT and CIBMTR meeting.

Alison Betof Warner, MD, PhD, assistant attending physician, Memorial Sloan Kettering Cancer Center, discussed the solid tumor session she chaired at the 2023 Tandem Meetings.

Samiah Al-Zaidy, MD, vice president of clinical development and lead on the GM1 Program at Passage Bio, discussed the latest results from the Imagine-01 clinical trial evaluating the investigational treatment PBGM01.

The associate professor of neurology at Columbia University spoke about the session he will be chairing at MDA’s 2023 conference.

The associate professor of medicine at Sylvester Comprehensive Cancer Center discussed updated data from the phase 3 ALLELE study.

Four of 5 patients showed improvements in ejection fraction following bone marrow transplant.