News

July 7, 2020July 7, 2020

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

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 ... Read more

June 30, 2020June 30, 2020

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

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. ... Read more

June 23, 2020July 28, 2020

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

News, syndicated

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 ... Read more

June 23, 2020June 23, 2020

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

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 ... Read more

June 16, 2020June 24, 2020

Higher Daily Doses of OV101 Could Lessen Benefits via Changes in Nerve Cells

News

Note: This story was updated on June 24, 2020, to clarify that higher daily doses, rather than long-term exposure, were thought to contribute to the loss of benefit. The ... Read more

June 10, 2020June 10, 2020

Coalition Will Address Racial Disparities in Rare Disease Communities

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 ... Read more

June 2, 2020June 2, 2020

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

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 ... Read more

May 28, 2020May 28, 2020

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

News, syndicated

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 ... Read more

May 26, 2020May 26, 2020

AskBio and UNC Team Up to Develop Angelman Syndrome Gene Therapy

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 ... Read more

May 25, 2020May 25, 2020

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

News, syndicated

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 ... Read more

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