Angelman Syndrome News

July 28, 2020July 29, 2020

Ovid, UConn Collaborating to Develop shRNA Gene Therapy for Angelman

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 14, 2020July 15, 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

July 9, 2020August 9, 2020

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

Angelman Syndrome News

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 in ... Read more

July 7, 2020July 7, 2020

CASS, ASF Team Up to Support Angelman Families, Increase Research

Angelman Syndrome News

The Angelman Syndrome Foundation (ASF) and the Canadian Angelman Syndrome Society (CASS) are partnering, both to increase research into the complex neurological genetic disorder and to support the families affected ... Read more

June 30, 2020July 1, 2020

Common Genetic Variation May Be Tied to Imprinting Defects, Study Finds

Angelman Syndrome News

The presence of a common genetic variation in women may increase the risk of deficient genomic imprinting and the development of Angelman syndrome in their children, a study shows. The ... Read more

June 23, 2020July 24, 2020

Alexion Charitable Foundation Giving $1.1M to Assist Rare Disease Community During Pandemic

Angelman Syndrome News, COVID-19 News

The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during the COVID-19 pandemic, the organization recently announced. The grants will support ... Read more

June 23, 2020June 23, 2020

OV101 Gets FDA’s Rare Pediatric Disease Status for Treating Angelman

Angelman Syndrome News

Ovid Therapeutics‘ OV101 (gaboxadol) has been granted a rare pediatric disease designation by the U.S. Food and Drug Administration (FDA) for the treatment of Angelman syndrome. This designation is given to experimental ... Read more

June 16, 2020June 17, 2020

Higher Daily Doses of OV101 Could Diminish Benefits, Evidence Suggests

Angelman Syndrome News

Long-term exposure to Ovid‘s treatment candidate for Angelman syndrome patients, OV101 (gaboxadol), may result in adaptive changes in nerve cells that cause a loss of therapeutic benefit over time, new ... Read more

June 10, 2020July 11, 2020

Coalition Will Address Racial Disparities in Rare Disease Communities

Angelman Syndrome News, COVID-19 News

The Black Women’s Health Imperative (BWHI) recently created a Rare Disease Diversity Coalition focused on reducing racial disparities in the rare disease community. Getting a timely and accurate diagnosis for ... Read more

June 2, 2020June 2, 2020

FAST Awards $3M to UC Davis Scientists Seeking Better AS Treatments

Angelman Syndrome News

Supporting efforts to find effective treatments for Angelman syndrome (AS), the Foundation for Angelman Syndrome Therapeutics (FAST) has awarded two UC Davis MIND Institute scientists a $3 million grant. The recipients are ... Read more

May 28, 2020June 28, 2020

COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

Angelman Syndrome News, COVID-19 News

While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has begun to cause delays in the development of gene therapies ... Read more

May 26, 2020May 26, 2020

AskBio and UNC to Develop Angelman Syndrome Gene Therapy

Angelman Syndrome News

Asklepios BioPharmaceutical (AskBio) and the University of North Carolina at Chapel Hill (UNC) have entered into an agreement for the development of a gene therapy for Angelman syndrome. Angelman syndrome ... Read more

May 25, 2020June 25, 2020

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

Angelman Syndrome News

European authorities must step up efforts to screen babies for a multitude of genetic disorders, a panel of experts suggested during a May 14-15 online medical conference. The session was ... Read more

May 21, 2020June 23, 2020

Video Games Connect Chronically Ill Children Isolated at Home, Hospital

Angelman Syndrome News, COVID-19 News

Dara Riva always had a rule that her 10-year-old son could play video games only once a week. But then the COVID-19 pandemic struck, and her perspective changed. Riva’s son, ... Read more