News

March 21, 2019March 21, 2019

With Improvements, ‘DriveSafe DriveAware’ Software Can Help Assess Huntington’s Patients’ Fitness to Drive, Study Shows

News

A software known as “DriveSafe DriveAware” can help evaluate if patients with Huntington’s disease still have the cognitive fitness needed to safely drive a car, a pilot study shows. ... Read more

March 19, 2019March 19, 2019

Biomarker Platform That May Track Huntington’s Decades Before Symptom Onset Receives FDA Letter of Support

News

The U.S. Food and Drug Administration (FDA) has issued a letter of support encouraging sRNAlytics to further develop its sRNA-FIND platform for discovering and monitoring variants of small RNA molecules that ... Read more

March 14, 2019

Brexit Could Have Real Effects for UK Rare Disease Patients, Experts Warn

News

Madeline Collin, a 24-year-old activist with Gaucher disease, worries that patients like her will suffer deeply if Britain leaves the European Union (EU), as scheduled, at the end of ... Read more

March 14, 2019March 14, 2019

Mini Pig Model May Be Useful for Assessing Huntington’s Symptoms, Progression, Study Suggests

News

Analysis of behavior patterns in genetically engineered mini pigs could be a valuable tool for assessing Huntington’s disease symptoms, a study shows. This new animal model may help researchers ... Read more

March 12, 2019March 12, 2019

MP101 Given FDA’s Orphan Drug Status for Potential to Treat Huntington’s Disease

News

The U.S. Food and Drug Administration has granted orphan drug designation to MP101, an investigational mitochondrial-targeted neuroprotective agent, to treat Huntington’s disease. Mitochon Pharmaceuticals, the therapy’s developer, expects to begin a Phase ... Read more

March 12, 2019March 11, 2019

Moral Dilemmas Complicate Treatment of Rare Diseases, Says Israeli Bioethicist

News

With each new advance in medicine comes ethical dilemmas, from fertility treatments and newborn screening, to vaccinations, gene therapies and euthanasia. But rare diseases and the expensive therapies needed ... Read more

March 7, 2019March 7, 2019

Gene Therapy Candidate AMT-130 Shows Promise in Preclinical Findings

News

Encouraging new preclinical data on AMT-130, uniQure’s experimental gene therapy candidate for Huntington’s disease, were presented at the 14th Annual Huntington’s disease Therapeutics Conference. The conference was organized by ... Read more

March 5, 2019March 5, 2019

NIH Rare Disease Day Highlights Joint Networks Advancing Array of Research

News

Rare diseases affect about 30 million Americans — roughly the same number as those with type 2 diabetes. Yet only 5 percent of the estimated 7,000 rare diseases known ... Read more

February 28, 2019February 28, 2019

Tiny Chip Will Speed Up Genetic Diagnosis of Huntington’s Disease

News

Researchers have developed a miniature device that can measure the length of disease-related DNA regions in less than five minutes. The technology was tested using DNA samples from myotonic ... Read more

February 26, 2019February 25, 2019

WODC 2019 Organizers Expect 1,200 to Attend Rare Disease Conference in April

News

The world’s biggest gathering of rare disease researchers, patient groups, pharmaceutical executives, and government officials is planned for April 10–12 in a Washington, D.C., suburb. Some 1,200 people have ... Read more

Pin It on Pinterest