News

October 7, 2019October 7, 2019

Windy City Angels Set to Run Chicago Marathon to Support Angelman Syndrome Foundation

News

Whether by donating or cheering on the sidelines, people can show their support for the running team put together by the Angelman Syndrome Foundation (ASF) during the Bank of ... Read more

September 30, 2019September 30, 2019

Israeli Angelman Foundation Works to Give ‘Strength’ and ‘Answers’ to Struggling Families

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From the balcony of their spacious home in the Israeli town of Beit Shemesh, Faige and David Spolter enjoy a commanding view of the Judaean Hills, and the ancient ... Read more

September 26, 2019

NORD 2019 Rare Disease Summit Set for Oct. 21-22 in Washington, DC

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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 ... Read more

September 23, 2019October 2, 2019

Phase 3 Trial of OV101, Possible Angelman Therapy, Randomizes 1st Patient

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A first patient has been randomized in the NEPTUNE trial, which is testing oral OV101 (gaboxadol) as a potential treatment for Angelman syndrome. Ovid, the pharmaceutical company that is developing ... Read more

September 23, 2019September 23, 2019

Israeli Angelman Syndrome Clinic Earns Global Respect

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Israel’s Sheba Medical Center at Tel HaShomer already ranks as one of the world’s 10 best hospitals, according to Newsweek magazine. Now, the institution has a new accolade: it’s the ... Read more

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

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