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Top Gaucher Disease News News

Wearable and Flexible Electrochemical Sensors for Sweat Analysis: a Review

Parkinson's Disease · China

Wearable and Flexible Electrochemical Sensors for Sweat Analysis: a Review

75% Left coverage: 4 sources

Flexible wearable sweat sensors allow continuous, real-time, noninvasive detection of sweat analytes, provide insight into human physiology at the molecular level, and have received significant attention for their promising applications in personalized health monitoring. Electrochemical sensors are the best choice for wearable sweat sensors due to their high performance, low cost, miniaturization, and wide applicability. Recent developments in s…See the Story

On teaching my children the benefits of volunteering

Volunteering · Alaska

On teaching my children the benefits of volunteering

Last Saturday, my 7-year-old daughter, Violet, and I volunteered with Stream Watch, an award-winning program that takes care of high-use fishing sites on Alaska’s Kenai Peninsula. Stream Watch volunteers pick up litter, collect monofilament from recycling tubes, teach visitors about ethical angling, provide natural history education, and promote bear safety. This mighty cadre removes hundreds of pounds of trash, connects with hundreds of both ne…Read Article

10 strategies that help us survive medical travel and appointments

Anchorage, United States · Anchorage

10 strategies that help us survive medical travel and appointments

To manage the stress and chaos of medical travel for her young daughter, columnist Marion Glaser prioritizes fun, family, and friends.Read Article

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.Maryland · Maryland Read Article

My unexpected diagnosis with Gaucher disease type 1

When most people imagine being diagnosed with a disease, they picture a cold, sterile doctor’s office and a somber physician delivering bad news. But my story didn’t start like that. I was a healthy kid with no red flags, no strange symptoms, and no reason to think I might have a genetic disease. My diagnosis with Gaucher disease in 2020 came completely out of the blue, triggered by my sister’s prenatal genetic testing. I found out by walking in…Read Article

Gaucher disease gene therapy FLT201 shows sustained clinical benefits

Treatment with the experimental gene therapy FLT201 led to sustained clinical benefits that lasted up to 21 months for people with Gaucher disease type 1, according to new clinical trial data. Spur Therapeutics, the therapy’s developer, presented the findings at the 28th annual meeting of the American Society of Gene and Cell Therapy (ASGCT) in New Orleans. “These findings highlight the power of our approach to provide gene therapies that set ne…Maryland · Maryland Read Article

Monitoring lyso-Gb1 may aid decision to start ERT in Gaucher disease: Study

Measuring levels of glucosylsphingosine (lyso-Gb1), a diagnostic biomarker of Gaucher disease, in children confirmed by newborn screening programs to have the disease may aid a decision about when to start enzyme replacement therapy (ERT). That’s according to a study in Italy that measured lyso-Gb1 levels monthly in three newborns with genetically confirmed Gaucher. At up to 20 months, or almost two years, the children didn’t have disease sympto…Italy · Italy Read Article

No safety concerns seen with VPRIV self-administration: Study

VPRIV (velaglucerase alfa), an enzyme replacement therapy (ERT) for Gaucher disease, can be safely administered by patients or caregivers without posing additional safety risks relative to infusions administered by a healthcare provider, a study showed. ERT self-administration was also safe in people with Fabry disease who received agalsidase alfa, which is sold as Replagal in some countries but isn’t approved in the U.S. Both drugs are marketed…United Kingdom · United Kingdom Read Article

Gene therapy eases neurological issues in Gaucher disease mouse model

A gene therapy designed to simultaneously deliver a healthy version of the GBA1 gene and the GDNF gene to cells in the brain eased neurological problems, prevented nerve cell death, and extended survival in a mouse model of Gaucher disease, a study shows. The animals were designed to mimic neuronopathic Gaucher disease, which refers to forms of the disease marked by neurological complications. While treatments are available to address other symp…United States · United States Read Article

Study: Cerdelga shows benefits in children with Gaucher disease type 1

Treatment with Cerdelga (eliglustat), a substrate reduction therapy approved in the U.S. for adults with type 1 Gaucher disease, was safe and showed therapeutic potential in children with the condition, a real-world study finds. “This case series highlights real-world experience with [Cerdelga] in pediatric [Gaucher type 1] patients, providing important insights into its potential to improve clinical outcomes and optimize disease management,” th…United States · United States Read Article

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