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Gene-Editing Therapy Promising for Rare Hereditary Disease
A gene-editing therapy led to durable changes in serum transthyretin levels among people with hereditary transthyretin ...
Breakthrough Therapy designation granted based on FDA’s review of available clinical evidence of safety and efficacy from all ...
One oral presentation and five poster presentations will highlight exciting progress in AskBio’s pipeline and advancements in ...
French biotech EG 427 has had success with its urinary incontinence DNA therapy, EG110A, showing a more than 80% reduction in ...
Treatment introduces an expansion of the scope of gene therapies that might lead to similar treatments for neurodegenerative ...
How cell and gene therapy developers can lessen the chances of receiving FDA complete response letters through the use of ...
Taysha Gene Therapies' investigational treatment for Rett syndrome has received breakthrough-therapy designation from the Food and Drug Administration.
In a preclinical study, UC Davis Comprehensive Cancer Center scientists have developed a highly targeted gene therapy that ...
The FDA’s Breakthrough Therapy designation, which expedites development and regulatory review, was granted based on clinical evidence from Part A of the REVEAL Phase 1/2 trials. The data showed a 100% ...
The FDA has granted Fast Track designation to SAR446268 for the treatment of non-congenital myotonic dystrophy type 1.
By Daniella Parra NanoCell Therapeutics, Inc. has appointed three new members to its Scientific Advisory Board: Prof. Zoltán ...
After years of heartbreak, researchers have found an experimental treatment that can slow the progression of Huntington’s ...
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