An expert involved in the first successful gene therapy trial for Huntington's Disease discusses the trial results and challenges with the FDA.
Gene therapy represents one of medicine’s most ambitious attempts to treat disease at its root cause by altering the genetic code itself. The approach works by recovering the functions of critical ...
Chantez Sanford Jr., 24, of Southfield, says his life has been transformed after undergoing Lyfgenia gene therapy at Children ...
SEATTLE — Some of our most challenging health conditions result from genetic disorders like sickle cell disease and certain cancers. Gene therapy is revolutionizing how they're treated, and the Fred ...
In a world-first trial, scientists used a one-off CRISPR gene edit to switch off a liver “fat brake” gene, slashing stubborn LDL cholesterol and triglycerides in patients whose levels refused to budge ...
The US Food and Drug Administration (FDA) has fast-tracked a promising new gene therapy, KB707, which is inhaled as a fine mist for the targeted treatment of advanced or metastatic non-small-cell lung ...
Zacks Investment Research on MSN
SRPT stock up as enrollment begins in DMD gene therapy safety study
Shares of Sarepta Therapeutics SRPT rose about 2% yesterday after it announced that screening and enrollment are underway in ...
A new method for safely inserting large chunks of DNA into genomes has now measured up in mice, potentially paving the way ...
Enh3ance trial results indicate DTX301 reduces ammonia levels by 18% and supports dietary liberalization in patients with OTC deficiency.
Solid Biosciences’ SGT-003 is the only late‑stage program to show early cardiac benefit across biomarkers and function, according to William Blair.
After telegraphing an investigation in November, the FDA has moved to restrict the use of bluebird bio’s gene therapy Skysona in certain patients. The FDA has updated Skysona’s indication, allowing it ...
Some results have been hidden because they may be inaccessible to you
Show inaccessible results