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August 13, 2019August 13, 2019

Huntington’s Patients Have Difficulty Recognizing Mental States of Others, Study Reveals

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Patients at a pre-symptomatic stage of Huntington’s disease have marked difficulties recognizing and understanding negative and neutral mental states of other individuals, regardless of gender, a study has found. ... Read more

August 8, 2019August 8, 2019

HD Patients Prefer Less Invasive, No-Placebo Gene Therapy Trials, Survey Says

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Eligible Huntington’s disease patients would likely participate in a gene therapy trial regardless of the study design, but they are more inclined toward those with less invasive interventions and ... Read more

August 6, 2019August 6, 2019

Shape Changes in Neostriatum Linked to Clinical Features of Huntington’s

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Changes in the shape of a brain region called the neostriatum — involved in motor and cognitive control — are associated with specific clinical features in people with Huntington’s ... Read more

August 1, 2019

Rare Disease Groups Seek Public Support to Renew Newborn Screening Act in Senate

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Screening newborns for genetic diseases with treatments that can prevent crippling or deadly progression, especially for rare disorders, has a ways to go in the United States. No state ... Read more

August 1, 2019August 1, 2019

Stem Cell Transplants Using Nasal Delivery Show Promise in Huntington’s Mouse Model

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Stem cell therapy delivered via the nose led to longer lives and evidence of better circadian (internal “clock”) activity in a mouse model of Huntington’s disease, and may offer ... Read more

August 1, 2019

Oklahoma Ranks Lowest on Programs Key to Rare Diseases on NORD’s 2019 State Report Card

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Oklahoma suffers more tornadoes than any other state, has the highest per-capita rate of women in U.S. prisons, ranks second in the number of teen births per 100,000 teenage ... Read more

July 30, 2019July 30, 2019

Early Data Show Neflamapimod’s Potential to Prevent Nerve Cell Degeneration

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Treatment with EIP Pharma’s investigational therapy neflamapimod (VX-745) can restore nerve cells’ communications and prevent their progressive degeneration and death, new preclinical results show. All together these data further ... Read more

July 25, 2019July 25, 2019

Cdk5 Protein Activity in Brain Linked to Depression, Potentially Even Treatment, in Huntington’s, Mouse Study Suggests

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Differences in the activity of a protein called Cdk5 in the brain could be a cause of — and perhaps even a target for the treatment of — depression ... Read more

July 23, 2019July 23, 2019

Phase 2 Trial Recruiting Early-stage Huntington’s Patients to Test Neflamapimod for Improving Cognition

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EIP Pharma is launching a proof-of-concept Phase 2 clinical trial to assess the ability of therapeutic candidate neflamapimod to treat cognitive dysfunction in patients with early-stage Huntington’s disease. The ... Read more

July 18, 2019July 18, 2019

Cannabinoid Derivatives Show Therapeutic Potential in Mouse Model, But EHP-102 Still Superior

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Emerald Health Pharmaceuticals announced that two of its cannabinoid-derived candidates — CBGA-Q and CBGA-Q-Na Salt — showed anti-inflammatory and neuroprotective effects in a mouse model of Huntington’s disease. However, ... Read more

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