March 12 (Reuters) - Ultragenyx Pharmaceutical said on Thursday its experimental gene therapy helped patients in a late-stage trial to better control ammonia levels linked to a rare inherited disorder ...
Regenxbio has guided its Duchenne muscular dystrophy (DMD) gene therapy candidate through another test, reporting a clean ...
After the FDA’s first-ever public listening meeting on data-sharing in the cell and gene therapy space, new draft guidance aims to standardize the practice. But recent decisions call into question ...
Bayer has discontinued an early-stage clinical gene therapy for a rare genetic disorder in favor of a similar candidate. | Bayer has discontinued an early-stage clinical gene therapy for a rare ...
A new chapter is unfolding for gene therapies in Europe. With retinal programs advancing into Phase I/II trials and regulators introducing more flexible approval pathways for rare diseases, the field ...
A pioneering gene therapy could help treat a rare seizure disorder called Dravet syndrome, according to new clinical trial ...
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Penn medical student who is genetic carrier of rare form of ALS on mission to develop gene therapy
Yentli Soto Albrecht, who is a genetic carrier of ALS, is working on research at Penn that could one day prove life-saving.
Add Yahoo as a preferred source to see more of our stories on Google. Biogen said the trial touted the once-yearly dosing potential of the ASO. Credit: Tada Images / Shutterstock.com (Tada Images / ...
A senior FDC official said the product is a “failed therapy” and confirmed that regulators have asked uniQure to run a new trial to prove the therapy actually helps Huntington’s patients. The ...
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First successes in the development of a gene therapy for incurable LAMA2-related muscular dystrophy
Researchers at the University of Basel have developed a gene therapy that could potentially treat a rare and currently fatal ...
Drug delivery researchers have vastly improved the potential of genetic therapies by overcoming the challenge of consistently getting genes and gene-editing tools where they need to be within cells.
UCLA researchers use lipid nanoparticles to insert full CFTR gene into airway cells, restoring function and opening new avenues for CF therapy.
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