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Selecting Right Mouse Model Key for Huntington’s Research, Therapy Development, Study Finds

Certain aspects of Huntington’s disease (HD), such as the biologic mechanisms of depression, are not consistently replicated in mouse models of the disorder, which may hinder therapy development, a study reports. The study, “Increased Serotonin Transporter Expression in Huntington’s Disease Patients Is Not Consistently Replicated in Murine Models,”…

Study Finds Abnormal Neural Development May Contribute to Huntington’s Disease, Offering New Therapeutic Strategy

Abnormal development of neural tissues may precede neurodegeneration in early-onset juvenile forms of Huntington’s disease, a recent study has found. Researchers also showed how targeting the cellular pathway involved in this process might be an effective strategy to restore normal neural development. Their study, “Expanded huntingtin CAG…

Gene Therapy Takes Center Stage at 2019 NORD Summit

With so much recent publicity surrounding gene therapy, it’s no surprise that the topic was a major focus of the recent 2019 NORD Rare Diseases & Orphan Products Breakthrough Summit. From diagnosis and clinical trial design to manufacturing, pricing strategies, and ethical concerns, gene therapy — both its high…