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August 12, 2019August 12, 2019

Spaced Training Can Improve Learning in Down, Angelman Patients, Mouse Study Shows

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Spaced training interventions may be used to minimize learning impairments associated with neurological disorders, including Down and Angelman syndrome, a mouse study has found. The results, “Spaced training improves ... Read more

August 5, 2019August 5, 2019

Behavioral Tests Useful in Assessing Memory and Motor Abilities in Angelman Children, Study Suggests

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Behavioral tests based on how well a child performs specific tasks may be promising tools for evaluating memory and motor abilities in those with Angelman syndrome (AS) and be of ... 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, 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 29, 2019July 29, 2019

Cannabidiol Reduces Seizures, EEG Abnormalities in AS Mice, Study Finds

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Treatment with cannabidiol reduces seizures and abnormal electric brain activity in mice with Angelman syndrome (AS), supporting its use as an anti-seizure treatment for patients, a study finds. The ... Read more

July 22, 2019July 22, 2019

Ovid’s OV101 Earns Orphan Drug Designation in Europe to Treat Angelman Syndrome

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The European Commission (EC) has granted orphan drug designation to Ovid Therapeutics’ OV101 for the treatment of Angelman syndrome. The therapy is being tested in the Phase 3 NEPTUNE ... Read more

July 18, 2019

European Initiative Targets Diagnosis, Treatment of Rare Diseases

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A new international consortium based in Paris, and funded largely by the 28-member European Union, intends to speed the diagnosis of rare diseases, while also accelerating the development of ... Read more

July 15, 2019July 15, 2019

Anxiety Common in Older Children and Especially Evident When ‘Preferred’ Caregiver Absent, Study Says

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Anxiety, especially separation distress, is very common among older children with Angelman syndrome and should be taken into consideration by doctors to improve both their quality of life and ... Read more

July 8, 2019July 8, 2019

Ovid Starts Phase 3 Trial of Experimental Therapy for Kids with Angelman

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Ovid Therapeutics has initiated the pivotal Phase 3 NEPTUNE trial to evaluate the experimental small molecule OV101 for the treatment of Angelman syndrome. The trial expects to enroll approximately ... Read more

July 5, 2019July 4, 2019

World’s First Alport Stamp Is Macedonian Mom’s Latest Win for Rare Disease Patients

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It wasn’t until Gordana Loleska’s son David was 14 years old that doctors in their native North Macedonia diagnosed his kidney, vision, and hearing problems as Alport syndrome. Although ... Read more

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