News

October 27, 2020October 28, 2020

UK Organization ‘Same But Different’ Announces ‘Glimmer of Hope’ Photo Contest Winner

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A photo of a bespectacled young boy, his red baseball cap slightly askew as he enjoys time outside, will be featured on the front cover of an upcoming calendar ... Read more

October 20, 2020October 20, 2020

Telehealth ‘Helpful’ Alternative to In-person Care, Rare Disease Patients Say

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A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a ... Read more

October 13, 2020October 13, 2020

Iron Therapy May Improve Sleep Quality in Young Angelman Patients

News

Sleep disturbances in people with Angelman syndrome may be at least partly associated with iron deficiency, according to a small study in the U.S. The data also showed that ... Read more

October 6, 2020October 6, 2020

Analysis Examines Health-related Quality of Life in Angelman Adolescents, Adults

News

Adolescents and adults with Angelman syndrome have a poor overall health-related quality of life (HRQoL) compared to the general population in the U.S., according to an exploratory analysis of ... Read more

October 6, 2020October 6, 2020

Same But Different Asks Public to Vote on ‘Glimmer of Hope’ Calendar Photos

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Same But Different, a nonprofit U.K. group that uses art for social change, is inviting people to choose their favorite photographs in a calendar contest to heighten awareness of rare ... Read more

September 29, 2020September 29, 2020

Neuren Pharmaceuticals Manufacturing NNZ-2591 for Upcoming Clinical Trials

News

Neuren Pharmaceuticals has begun manufacturing of NNZ-2591, an investigational treatment for Angelman syndrome and other diseases, for use in Phase 2 clinical trials. NNZ-2591 is an analogue of cyclic glycine proline ... Read more

September 22, 2020September 22, 2020

Healx Becomes FAST Partner to Repurpose Medicines for Angelman

News

Healx and the Foundation for Angelman Syndrome Therapeutics (FAST) are partnering to identify new therapies for Angelman syndrome from among already-approved and available medications, according to a company press ... Read more

September 15, 2020September 15, 2020

Lixte Teams Up With FAST to Support Preclinical Studies of LB-100

News

Lixte Biotechnology Holdings is teaming up with the Foundation for Angelman Syndrome Therapeutics (FAST) to advance preclinical studies investigating its proprietary lead compound, LB-100, as a potential treatment for ... Read more

September 8, 2020September 8, 2020

Parents: Cure for Angelman Should Be Priority of Gene Therapy Trials

News

Parents of children with Angelman syndrome think gene therapy trials to find a cure should be a priority and should focus on neurological symptoms as well as communication and motor ... Read more

September 1, 2020September 1, 2020

Same But Different Photo Contest Celebrates People With Rare Diseases

News, syndicated

Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for rare diseases. Under the theme ... Read more