Gaucher disease gene therapy FLT201 shows proof of concept
FLT201, a gene therapy for Gaucher disease type 1, led to sustained increases in enzyme levels in animal models, a study showed.| Gaucher Disease News
Learn about the various symptoms of Gaucher disease, including those that are bone- or organ-related, and neurological symptoms.| Gaucher Disease News
Enzyme replacement therapy (ERT) is a key treatment approach for Gaucher disease that addresses the underlying cause of the genetic disorder.| Gaucher Disease News
FLT201, a gene therapy for Gaucher disease type 1, led to sustained increases in enzyme levels in animal models, a study showed.| Gaucher Disease News
Gaucher disease patients are at higher risk of blood cancer, but the risk of solid tumors is similar to the general population, per a study.| Gaucher Disease News
International Gaucher Day on Oct. 1 kicks off this year's awareness month, with a goal of shortening the diagnostic journey for patients.| Gaucher Disease News
International Gaucher Day on Oct. 1 kicks off this year's awareness month, with a goal of shortening the diagnostic journey for patients.| Gaucher Disease News
Columnist Rivka Silver has found that strength training with Pilates has been the exercise that works best to relieve her inflammation.| Gaucher Disease News
Treatment with a corticosteroid and hydroxychloroquine eased symptoms of severe lung problems in an infant with type 3 Gaucher disease.| Gaucher Disease News
A boy with Gaucher disease developed digestive complications related to lymph node issues despite receiving ERT treatment, per a case report.| Gaucher Disease News
Casma Therapeutics will develop CSM-101 as a potential oral treatment for Parkinson's disease and Gaucher disease.| Parkinson's News Today
