4D Molecular Therapeutics’ 4D-310 effected clinically meaningful improvements in left ventricular function, exercise capacity, and quality of life.
Lysosomal Disorders
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All dose-escalation patients withdrew from enzyme replacement therapy (ERT) and remain off ERT.
CGTLive takes a look at some upcoming FDA decisions for Rare Disease Day.
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.
Ultragenyx’s self-complementary AAV9-based gene therapy reduced levels of relevant CSF biomarkers, as well as improvements in neurocognitive assessments and behavioral domains, among patients without neurodegeneration.
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.
Most treated participants were within 2 SDs of normative mean in acquiring cognition, expressive language and fine motor skills.
Interim data from the phase 1/2 CAMPSIITE trial were presented at WORLDSymposium 2023.
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.
Review top news and interview highlights from the week ending February 24, 2023.
Long term data from 2 clinical trials and expanded access programs were presented at WORLDSymposium 2023.
The patient has not re-started any of his previous Gaucher-specific therapy since receiving AVR-RD-02.
Two patients treated with AVR-RD-02 showed clinically meaningful reductions in liver size.
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.
The locum consultant of pediatric BMT and leukemia at Royal Manchester Children’s Hospital discussed the limited distribution model of arsa-cel in Europe.
No adverse events related to RP-L201 have been reported to date.
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.
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.
Mesoblast is resubmitting its BLA with new CMC, survival, and mechanism of action data on the therapy and phase 3 trial.
Olga Uspenskaya, MD, PhD, vice president, clinical development, Prevail Therapeutics, discussed the PROCEED trial of PR001.
The professor of pediatrics at Stanford Cancer Institute discussed recent data from clinical trials of the lentiviral gene therapy RP-L102.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
