Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Lysosomal Disorders
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Statistically significant improvements in cognitive development were only seen in the cohort of patients younger than 30 months.
The gene therapy was approved in the European Union earlier this year.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
The first 3 patients dosed have been attack-free for up to 10.5 months.
The Genomics and Epigenetic Guided Safe Harbor mapper will aid in the future design of gene-editing therapies.
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 November 4, 2022.
Abeona has announced topline results from its phase 3 VIITAL study of EB-101.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
The pediatrician from Royal Manchester Children’s Hospital discussed how further research in Gaucher disease can benefit the field of rare lysosomal diseases as a whole.
Review top news and interview highlights from the week ending October 28, 2022.
CGTLive highlights the PROVIDE trial of PR001 for Gaucher Awareness Month.
AVR-RD-02 also received ILAP designation in the UK earlier this month.
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
The pediatrician from Royal Manchester Children’s Hospital discussed progress with the PROVIDE trial of PR001.
The trial is being conducted at the University of California – San Diego under the leadership of Stephanie Cherqui, PhD.
Rossano discussed unmet needs for patients with Danon disease and the potential of investigational gene therapy RP-A501.
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.
Pam Gavin, executive vice president of NORD, discussed actions the organization is taking to further improve access to advanced care.
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.
With so many therapies on the horizon, the clinical and patient communities need to be adequately prepared to support them.
Participant 3 showed an 89% decrease in NAA in the CSF at 3 months post-treatment.
