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September 16, 2019September 16, 2019

Foundation Honors 4 Individuals for their Contributions to Angelman Research, Patient Advocacy

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The Angelman Syndrome Foundation (ASF) recently honored four individuals for their exceptional contributions to the Angelman syndrome community and to advancing research. Awards were presented to a special education ... Read more

September 9, 2019September 9, 2019

Potential Angelman’s Treatment, GTX-102, Named Orphan Drug and Rare Pediatric Disease Therapy by FDA

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A potential treatment for Angelman syndrome, GTX-102, has been granted both orphan drug and rare pediatric disease designations by the U.S. Food and Drug Administration (FDA) to support and help speed ... Read more

September 2, 2019August 30, 2019

‘Disorder’ Film Festival Offers Look at Siblings Touched by Rare Diseases

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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: ... Read more

August 28, 2019August 28, 2019

At NY Genome Center, Legal Expert Presents Ethical Dilemmas in Gene Editing

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Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert Timo Minssen outlined the potentially explosive ethical landmines surrounding such ... Read more

August 26, 2019August 26, 2019

Blocking Two Proteins Linked to UBE3A Activity Might Treat Angelman, Study Suggests

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Lowering the levels of two proteins involved in nerve cell development and tightly linked to the activity of UBE3A — the enzyme that is faulty in Angelman syndrome — ... Read more

August 19, 2019August 19, 2019

Neuren’s Investigational NNZ-2591 Rescues Motor and Cognitive Functions, Mouse Study Shows

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Neuren Pharmaceutical’s candidate NNZ-2591 has shown positive effects in a mouse model of Angelman syndrome, treating all symptoms of the disease, the company announced. These results were used to ... Read more

August 19, 2019August 19, 2019

Nebraska’s Neena Nizar Seeks Cure for Jansen’s, One of World’s Rarest Diseases

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Imagine living your whole life with a painful disease so rare that only 25 others worldwide have what you have. And that you’re one of just six such people ... Read more

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

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