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
One-time treatment with the gene therapy FLT201 (avigbagene parvec) may help control Gaucher disease without the need for additional therapies, according to new data from four patients in an early clinical trial. Spur Therapeutics, the company developing FLT201, presented the data at the European Society of Gene and Cell Therapy (ESGCT) 32nd Annual Congress, held […] The post One-time gene therapy controls Gaucher disease symptoms appeared first on Gaucher Disease News.| 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
October 13, 2025| 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
October 15, 2025| Gaucher Disease News
My interest in nutrition began when I started noticing how much food affects how I feel — not just physically, but mentally and emotionally, too. Learning about the connection between diet, inflammation, and overall wellness opened my eyes to how much influence we have through the choices we make each day. Living with Gaucher disease, […] The post My journey with Gaucher disease inspired a new path forward appeared first on Gaucher Disease News.| 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
A bone marrow biopsy — a test that examines the soft tissue inside bones where blood cells are made — proved critical in diagnosing Gaucher disease (GD) in a boy in India with growth delays and a swollen abdomen. The biopsy revealed the presence of Gaucher cells (enlarged foamy immune cells that are a hallmark […] The post Bone marrow biopsy proves key to boy’s Gaucher diagnosis appeared first on Gaucher Disease News.| 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
August 27, 2025| Gaucher Disease News
Living with Gaucher disease has shaped how I think about my body in ways I never expected. For many people, body image is about appearance. For me, it has been less about the surface and more about the deeper, unsettling feeling that my body is failing me. When I was diagnosed with Gaucher, it came […] The post My Gaucher diagnosis made me feel like my body had betrayed me appeared first on Gaucher Disease News.| Gaucher Disease News
Measuring levels of lyso-Gb1, a fatty molecule often used as a biomarker of Gaucher disease (GD) severity, could help improve the accuracy of diagnostic tests for the rare condition, according to a new report from a South American research team. While low activity of the glucocerebrosidase (GCase) enzyme is the diagnostic hallmark of Gaucher, unreliable […] The post Measuring lyso-Gb1 levels may aid diagnosis accuracy in Gaucher appeared first on Gaucher Disease News.| Gaucher Disease News
A 31-year-old woman with type 1 Gaucher disease (GD) experienced worsening symptoms and rising disease activity markers after switching from long-term enzyme replacement therapy (ERT) to Cerdelga (eliglustat), according to a recent case report. The woman — who was obese and had her spleen surgically removed years earlier — developed increasing fatigue, liver enlargement, and […] The post Obesity may affect SRT treatment response in Gaucher disease appeared first on Gaucher Disease News.| Gaucher Disease News
As the Jewish new year approaches, I find myself in a season of reflection. I’m looking back on the past year and taking stock of how far I’ve come, while also looking ahead with hope and intention. Living with Gaucher disease means that I’m always aware of my body, my energy, and my limits. But […] The post Reflecting on how I’ve grown stronger over the past year appeared first on Gaucher Disease News.| Gaucher Disease News
In Gaucher disease, the toxic buildup of a fatty molecule called glucosylsphingosine (lyso-Gb1) leads to issues with how nerve cells generate energy and other cellular problems, according to a new study. Lyso-Gb1 is a biomarker of disease burden and response to treatment. The findings help to understand how the accumulation of fatty molecules contributes to […] The post Fatty molecule may cause nerve cell damage in Gaucher: Study appeared first on Gaucher Disease News.| 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
Gaucher disease is a genetic disorder that can cause enlarged organs, blood disorders, bone abnormalities, and neurological complications.| Gaucher Disease News
