Review top news and interview highlights from the week ending October 28, 2022.
Neurology
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CGTLive highlights the PROVIDE trial of PR001 for Gaucher Awareness Month.
The trial expects to enroll its first patient in the first half of 2023.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
As we learn more about genomics and identify more genes tied to rare disorders, the role of genetic counselors will become even more critical.
Review top news and interview highlights from the week ending October 21, 2022.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
The executive director of Hemophilia Foundation Southern California discussed issues with minorities accessing care for rare diseases and strategies to mitigate them.
The proportion of patients with concentrations of NfL and MBP above the upper limit of normal decreased from 67% and 63%, respectively, to 12% at 5 years.
With so many therapies on the horizon, the clinical and patient communities need to be adequately prepared to support them.
The director of the Regenerative Medicine Institute at Cedars Sinai discussed research being conducted with CNS10-NPC-GDNF in multiple indications.
Participant 3 showed an 89% decrease in NAA in the CSF at 3 months post-treatment.
Review top news and interview highlights from the week ending October 14, 2022.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Nearly 20% of treated patients achieved at least 25% improvement in timed 25-foot walk speed and/or 9-Hole Peg test at 28 weeks.
The director of the Regenerative Medicine Institute at Cedars Sinai discussed results from a phase 1/2a study of CNS10-NPC-GDNF.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Data from the first 3 of the 8 patients dosed indicates that the patients improved on the GMFM and the MSEL.
Solid has also pivoted from developing SGT-001 to SGT-003 for treating Duchene muscular dystrophy.
Review top news and interview highlights from the week ending September 30, 2022.
Rob Richards, administrative director, cell therapy and transplant, University of Pennsylvania, discussed improving reach of gene therapy trials and therapies.
SRP-9001 has shown safety and efficacy across multiple studies compared to controls, with additional efficacy data expected in 2024 from the EMBARK trial.
A phase 1 and phase 1b study of CK0803 will soon be initiated following the FDA’s IND clearance.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Although efficacy and HGF expression were assessed as outcome measures, comparisons were not able to be made between placebo and Engensis groups.
