Angelman Syndrome News

January 12, 2021January 13, 2021

ASF’s 15q Clinical Research Network Offers Q & A on COVID-19 Vaccines

As COVID-19 vaccination programs are being rolled out across the U.S., the Angelman Syndrome Foundation’s 15q Clinical Research Network (CRN) is seeking to address questions and concerns about the vaccines from ... Read more

December 15, 2020December 16, 2020

Angelman Children May Be More Prone to Cavities, Other Teeth Problems, Study Says

Angelman Syndrome News

Children with Angelman syndrome may be more prone to developing cavities in their teeth, due to the growth of thin enamel with an abnormal protein content, according to a new ... Read more

December 8, 2020December 9, 2020

OV101 Fails to Meet NEPTUNE Trial Goals; Ovid Will Suspend Development

Angelman Syndrome News

OV101 (gaboxadol), designed to improve motor skills, cognition, and memory in people with Angelman syndrome, did not meet its primary goal (endpoint) in Ovid Therapeutics’ Phase 3 NEPTUNE clinical trial, ... Read more

December 1, 2020December 2, 2020

CRISPR-Cas9 Gene Therapy Shows Promise in Preclinical Angelman Study

Angelman Syndrome News

A CRISPR-Cas9-based gene therapy designed to increase the production of ubiquitin protein ligase E3A (UBE3A) — the enzyme lacking in Angelman syndrome — partially or completely corrected physical and behavioral deficits in a mouse model ... Read more

November 26, 2020December 28, 2020

Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe

Angelman Syndrome News, COVID-19 News

The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their health and well-being, according to a ... Read more

November 24, 2020November 24, 2020

In-person Engagement Can Help Foster Imitation in Children With Angelman Syndrome

Angelman Syndrome News

In-person social engagement is more important for fostering imitative behaviors in children with Angelman syndrome than in typically-developing children, a new study suggests. The study, “Imitation in Angelman syndrome: the ... Read more

November 10, 2020November 11, 2020

GTX-102 Leads to Significant Improvements in Trial, But Also Causes Muscle Weakness

Angelman Syndrome News

Use of GTX-102, GeneTx Biotherapeutics and Ultragenyx’s investigational therapy for Angelman syndrome, led to substantial improvements in disease symptoms among children and adolescents across several treatment areas — including behavior, ... Read more

October 27, 2020November 28, 2020

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

Angelman Syndrome News

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 in ... Read more

October 13, 2020October 14, 2020

Iron Therapy May Improve Sleep Quality in Young Angelman Patients

Angelman Syndrome 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 supplementation ... Read more

October 6, 2020October 7, 2020

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

Angelman Syndrome 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 a ... Read more

October 6, 2020November 6, 2020

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

Angelman Syndrome News, COVID-19 News

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 diseases, ... Read more

September 29, 2020September 29, 2020

Neuren Pharmaceuticals Manufacturing NNZ-2591 for Upcoming Clinical Trials

Angelman Syndrome 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 (cGP), ... Read more

September 22, 2020September 22, 2020

Healx Becomes FAST Partner to Repurpose Medicines for Angelman

Angelman Syndrome 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 release. ... Read more

September 15, 2020September 15, 2020

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

Angelman Syndrome 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 Angelman ... Read more

September 8, 2020September 8, 2020

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

Angelman Syndrome 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 skills, ... Read more