News

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

August 25, 2020August 25, 2020

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

News, syndicated

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 ... Read more

August 18, 2020August 18, 2020

EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US

News, syndicated

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. ... Read more

August 11, 2020August 11, 2020

New Streaming Channel Showcases Rare Disease Films

News, syndicated

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 ... Read more

July 28, 2020July 28, 2020

Ovid, UConn Collaborating to Develop shRNA Gene Therapy for Angelman

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 ... Read more

July 21, 2020July 21, 2020

Angelini Pharma to Develop OV101 for Angelman in Europe

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 ... Read more

July 14, 2020July 14, 2020

Pinnacle Hill Project Aims to Treat Angelman by Changing Gene Activity

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 ... Read more

July 9, 2020July 9, 2020

National Survey Seeks to Assess Full Social, Economic Burdens of Rare Diseases in US

News, syndicated

The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease Survey to measure the full implications, economic and social, of living with rare disease ... Read more