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March 19, 2019March 19, 2019

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

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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

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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

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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

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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

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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

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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

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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

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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

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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

February 21, 2019February 21, 2019

Cannabinoid-Based Treatment for Huntington’s Granted Orphan Drug Designation by FDA

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tMMJ-002, a cannabis investigational product for the treatment of Huntington’s disease, has been granted Orphan Drug designation by the U.S. Food and Drug Administration, announced its developer MMJ International Holdings. This new status ... Read more

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