Among previously untreated adults with Gaucher disease type 1, a genetic disease in which there is improper metabolism due to a defect in an enzyme, treatment with the drug eliglustat resulted in ...
As gene therapies ramp up in spinal muscular atrophy and hemophilia, it may soon be the turn of Gaucher disease patients to realize the latent potential of this type of treatment. That’s according to ...
FLT201 is a highly differentiated AAV gene therapy candidate that delivers a longer-acting engineered variant of GCase, the enzyme missing in people with Gaucher disease Company expects to report ...
In 2010, we started noticing that my son Daniel had a bigger than normal belly. But we assumed that we were just doing a fine job of feeding him and were proud of that fact. This was until one day ...
LONDON, Sept. 09, 2021 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (Nasdaq: FRLN) (the “Company” or “Freeline”), a clinical-stage biotechnology company developing transformative ...
Spleen stiffness measurement (SSM) demonstrated strong predictive accuracy for liver decompensation in patients with compensated advanced chronic liver disease (cACLD), with an optimal cutoff of 50 ...
July 31 -- THURSDAY, July 30 (HealthDay News) -- Though its reputation doesn't rank down there with the appendix, the spleen isn't exactly known as a vital organ. In fact, plenty of people do fine ...
A rare genetic disorder that disproportionately affects Ashkenazi Jews is also a safeguard against tuberculosis, a new study shows, and that discovery could explain a longstanding scientific mystery.
Some results have been hidden because they may be inaccessible to you
Show inaccessible results
Feedback