Neurology

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.

An independent DSMB has also recommended continued enrollment in the trial’s first cohort.

The professor and director of the Sen. Paul D. Wellstone Muscular Dystrophy Specialized Research Center at University of Washington School of Medicine discussed the paper he recently coauthored.

Review top news and interview highlights from the week ending February 10, 2023.

Mehra and Subramanian discussed preclinical safety studies with the gene therapy SLS-004 and plans for future research.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Review top news and interview highlights from the week ending February 3, 2023.

Mehra and Subramanian discussed preclinical research with the investigational gene therapy SLS-004.

Cartesian Therapeutics’ Descartes-08 will be administered as 6 weekly infusions and does not require preconditioning chemotherapy.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

The dosing of additional patients in a double-blind, placebo-controlled design will help support a BLA submission for TSHA-120.

Olga Uspenskaya, MD, PhD, vice president, clinical development, Prevail Therapeutics, discussed the PROCEED trial of PR001.

Review top news and interview highlights from the week ending January 27, 2023.