Chantez Sanford Jr., 24, of Southfield, says his life has been transformed after undergoing Lyfgenia gene therapy at Children ...
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 ...
A 24-year-old Southfield college student has become the first patient in Michigan to receive a new gene therapy that could ...
UniQure and REGENXBIO are both dealing with FDA setbacks for their respective gene therapies, as regulatory experts question ...
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 ...
As newborn screening and rapid DNA sequencing become routine, we are poised to catch and treat inherited diseases at their earliest stages. Today, we can intervene in the first days or weeks of life.
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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 ...
Forget Vertex Pharmaceuticals -- CRISPR Therapeutics is a better buy right now.
Solid Biosciences’ SGT-003 is the only late‑stage program to show early cardiac benefit across biomarkers and function, according to William Blair.
A new method for safely inserting large chunks of DNA into genomes has now measured up in mice, potentially paving the way ...
RGX-121 was rejected in early February, with Regenxbio listing the primary reasons given by the FDA as concerns about the ability to properly define a patient population, the use of a natural history ...
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