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.
Neurology
Latest News
Advertisement
Advertisement
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
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.
Review top news and interview highlights from the week ending March 10, 2023.
The professor and director of the Sen. Paul D. Wellstone Muscular Dystrophy Specialized Research Center at University of Washington School of Medicine discussed working with the FDA toward a first disease-modifying therapy approval in Duchenne.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
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.
SNK01 is primarily being assessed in combination and as a monotherapy for treating solid tumors.
Competitors REGENXBIO and Capricor Therapeutics both have gene therapy candidates in clinical trials.
AskBio’s AB-1003 was previously granted fast track designation by the FDA.
Four of 5 patients showed improvements in ejection fraction following bone marrow transplant.
The therapy demonstrated an ability to repair injured muscle in mice models of Duchenne.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
4D Molecular Therapeutics’ 4D-310 effected clinically meaningful improvements in left ventricular function, exercise capacity, and quality of life.
All dose-escalation patients withdrew from enzyme replacement therapy (ERT) and remain off ERT.
In observance of Rare Disease Day, held annually on February 28, catch up on some of the latest data updates from clinical trials for rare diseases.
Patients who received the high dose of PBGM01 showed an increase in β-Gal activity in the CSF of 4.7 to 5.2 times baseline.
Al-Zaidy, the vice president of clinical development and lead on the GM1 Program at Passage Bio, discussed the results of the phase 1/2 Imagine-01 clinical trial of PBGM01.
The patient has not re-started any of his previous Gaucher-specific therapy since receiving AVR-RD-02.
FBX-101 showed promising efficacy and has been well-tolerated so far in the RESKUE trial.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
A third price model to be tested will limit the price of generic drugs for chronic conditions to $2 under Medicare plan D.
