Angelman Syndrome News

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

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

August 25, 2020August 29, 2020

Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input

Angelman Syndrome News

Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient input ... Read more

August 18, 2020August 18, 2020

EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US

Angelman Syndrome News

The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to support individuals with rare disorders who are pursuing personal goals through training and education. The ... Read more

August 18, 2020August 29, 2020

EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US

Angelman Syndrome News

The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to support individuals with rare disorders who are pursuing personal goals through training and education. The ... Read more

August 11, 2020August 29, 2020

New Streaming Channel Showcases Rare Disease Films

Angelman Syndrome News, COVID-19 News

When the COVID-19 pandemic forced the postponement of a rare disease film festival originally slated for May, its organizers set out to find a new way to bring the films ... Read more

July 28, 2020July 29, 2020

Ovid, UConn Collaborating to Develop shRNA Gene Therapy for Angelman

Angelman Syndrome News

Researchers at Ovid Therapeutics and the University of Connecticut (UConn) have announced a collaborative effort to expedite the development of a short hairpin RNA (shRNA) therapy for patients with Angelman syndrome. ... Read more

July 28, 2020August 29, 2020

Ovid, UConn Collaborating to Develop shRNA Gene Therapy for Angelman

Angelman Syndrome News

Researchers at Ovid Therapeutics and the University of Connecticut (UConn) have announced a collaborative effort to expedite the development of a short hairpin RNA (shRNA) therapy for patients with Angelman syndrome. ... Read more

July 21, 2020July 21, 2020

Angelini Pharma to Develop OV101 for Angelman in Europe

Angelman Syndrome News

Angelini Pharma has obtained development, manufacturing and commercialization rights to Ovid Therapeutics’ OV101 (gaboxadol), an investigational treatment for Angelman syndrome, in the European Union, Switzerland, Turkey, the U.K., and Russia. The partnership is ... Read more

July 21, 2020August 29, 2020

Angelini Pharma to Develop OV101 for Angelman in Europe

Angelman Syndrome News

Angelini Pharma has obtained development, manufacturing and commercialization rights to Ovid Therapeutics’ OV101 (gaboxadol), an investigational treatment for Angelman syndrome, in the European Union, Switzerland, Turkey, the U.K., and Russia. The partnership is ... Read more

July 14, 2020August 29, 2020

Pinnacle Hill Project Aims to Treat Angelman by Changing Gene Activity

Angelman Syndrome News

Pinnacle Hill will start a second project aimed at the development of a treatment for Angelman syndrome based on changing gene activity, the company announced. Pinnacle Hill is the result of ... Read more