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Caring for a loved one with a rare disease, especially during these uncertain times, demands significant time, attention, patience, and dedication. To help meet that need, the National Organization for Rare Disorders (NORD)’s Rare Caregiver Respite Program may be a helpful resource. The program seeks to give a…

Based on MRI scans, Huntington’s disease patients with major cognitive impairments have reduced gray matter volume and a thinner brain cortex than those without cognitive problems, a study has demonstrated.  Major cognitive decline was not associated with CAG repeat length, age, or education, suggesting additional…

U.K. Rare Diseases Framework, a government-led approach to raising national awareness of rare disorders, accelerating  diagnoses, and improving patient care and treatment, is now in practice. This  framework, signed by all four countries take make up the U.K. — England, Scotland, Wales and Northern Ireland — builds on the success…

The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences with rare diseases to speak at its upcoming virtual Living Rare, Living Stronger NORD Patient and Family Forum. The interactive, patient-focused forum will be held online June 26-27. The deadline to apply for…

Note: This story was updated Jan. 14, 2021, to note that pridopidine is no longer called Huntexil.  Treatment with pridopidine can slow the decline in total functional capacity in people with Huntington’s disease, a new analysis of clinical trial data indicate. The findings were published in the Journal…

The Huntington’s Disease Association of Ireland (HDAI) is spreading the word about a new documentary that tells the improbable story of Huntington’s disease (HD) families and their cross-continental journey to a Huntington’s gathering that included a meeting with Pope Francis. The recently released “Dancing at the Vatican” is…

Triplet Therapeutics announced it has completed patient enrollment in SHIELD HD, a natural history study documenting the course of Huntington’s in the absence of treatment and associated disease biomarkers. With more than 60 people taking part, SHIELD HD (NCT04406636) has attained and exceeded its target enrollment goal ahead…

A single injection of the protein CDNF — cerebral dopamine neurotrophic factor — into the brains of rats with induced Huntington’s disease protected affected nerve cells from degeneration and improved motor performance, a study reported. These findings support further development of CDNF as a treatment…

The first participant was dosed in a Phase 2a clinical trial testing Annexon’s investigational therapy ANX005 as a treatment for Huntington’s disease, the company announced. The open-label study ((NCT04514367) is currently enrolling patients at three sites in Washington state and Colorado; other sites across the U.S. are not yet…

To empower and equip members of the rare disease community to engage state leaders in matters of importance to patients and their families, the National Organization for Rare Disorders (NORD) has launched an initiative across the U.S. Its goal is to establish a Rare Disease Advisory Council (RDAC)…