News

Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a better time, says Marshall Summar, MD, chairman of NORD’s board of directors. “The pace of discovery in rare diseases has gone from brisk to hypersonic,” Summar told Bionews Services, publisher…

Laquinimod, an experimental immunomodulatory therapy developed by Active Biotech and Teva Pharmaceuticals, reduced signs of inflammation and brain atrophy (shrinkage) in patients with Huntington’s disease, data from an exploratory analysis of a Phase 2 trial show. The new data were presented by Active Biotech in three posters…

Huntington’s disease may manifest itself for the first time during neurulation, a process in which the neural tube — the precursor of our nervous system (brain and spinal cord) — develops while an embryo is forming, scientists report. These findings are in the study “Self-organizing neuruloids…

People with Huntington’s disease tend to use their own memories less in engaging with those around them, a new study found. The researchers say a diminished capacity for autobiographical recollection in these patients may prevent “the benefits of interpersonal bonding.” Titled “Discrete changes in the frequency…

Age of onset in Huntington’s disease is associated with a property of the inherited CAG repeat length in the huntingtin (HTT) gene — which determines the probability of further repeat expansions in nerve cells — rather than with the toxicity of the protein it produces, a study suggests. Along…

A small molecule that alters glucose metabolism in stressed brain cells may help prevent their death, representing a new strategy for treating brain diseases including amyotrophic lateral sclerosis, Alzheimer’s disease, and Huntington’s disease, among others. The potential therapeutic candidate was described in the journal Scientific Reports…

A new gene editing method allows researchers to target many different kinds of disease-causing mutations in multiple cell types, which could have implications for genetic disorders such as Huntington’s disease, a study suggests. The new method was described in the journal Cell Research in a study, titled “…

People at a pre-symptomatic, or premanifest, stage of Huntington’s disease (HD) have subtle speech alterations that may be used as a marker to evaluate new therapies intended to stop or slow disease progression before it becomes too debilitating. The study with that recent research, “…

Rare diseases deeply affect not only the children who experience them, but also their healthy brothers and sisters, as their parents can attest.    Two entries in November’s “Disorder: The Rare Disease Film Festival” will focus on what siblings go through, according to the San Francisco festival’s co-founder,…

Being less affected by loss is predictive of apathy in people with Huntington’s disease (HD), a new study has found. The study, “Insensitivity to Loss Predicts Apathy in Huntington’s Disease,” was published in the journal Movement Disorders. Apathy — the feeling of indifference, where a person just doesn’t care —…