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Gaucher Disease Insights
New report in partnership with Gaucher Community Alliance
New report in partnership with the Gaucher Community AllianceInclusion of Gaucher disease in newborn screening panels across the U.S.Written in partnership with the Gaucher Community Alliance, this report presents a compelling case for the inclusion of Gaucher disease in newborn screening panels across the United States. It highlights the clinical justification, technical feasibility, public health benefits, and cost-effectiveness of screening for Gaucher disease. Additionally, it examines state-level success stories and addresses common concerns to NBS for GD. View the Full ReportThe report is available for free online on the AllMyHealth website. www.allmyhealth.io/reports  | |||||||||
Top StoriesThree-year-old dies from Gaucher Disease due to delayed medicine. A child with Gaucher disease passed away after waiting months for necessary medication, sparking concern over healthcare delays and funding for such conditions. Read More →Data and Gene Therapy Transform Treatment Advances in gene therapy are changing how conditions like Gaucher disease are approached by targeting genetic roots, potentially offering more effective treatments with fewer lifelong management needs. Read More → | |||||||||
Latest ResearchResearchers have developed a new mouse model for chronic neuronopathic Gaucher disease, which exhibits features similar to Parkinson's disease. This model is crucial for understanding the complex interactions between Gaucher disease and neurological disorders like Parkinson's. The creation of this mouse model highlights the potential link between Gaucher disease and Parkinson's disease-like symptoms. By studying these models, researchers can gain insights into how Gaucher disease affects the nervous system and its relation to other neurodegenerative conditions, potentially leading to more effective treatments. This research underscores the importance of interdisciplinary approaches in understanding complex diseases, as noted by (Wen et al., 2025). | |||||||||
Clinical Trials This is a list of upcoming or ongoing clinical trials that are actively recruiting and have been listed or updated in the last two weeks: | |||||||||
Community News
Gaucher disease type 3 (also known as chronic neuronopathic Gaucher disease) has a later and more gradual onset compared with type 2. 
Attn: Gaucher Teens and Young Adults! - 
Our Voice Matters - First Virtual Meeting of Girls, Boys and Adolescents in Peru with Rare Diseases  | |||||||||
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