News

April 15, 2021April 15, 2021

EveryLife Scholarship Fund Open Until May 7 for Adults With Rare Diseases in US

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The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through training and education. For a ... Read more

April 14, 2021April 14, 2021

Ionis Plans to Launch Clinical Trial This Year for ION582

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Ionis Pharmaceuticals intends to launch a clinical trial later this year to test ION582, a potential Angelman syndrome therapy. The Phase 1/2a trial, designed to enroll up to 50 patients, ... Read more

April 7, 2021April 7, 2021

Cannabinoid BIO017 Receives FDA’s Orphan Drug Status

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The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to BIO017, a candidate therapy for Angelman syndrome being developed by Biom Therapeutics, the company has announced. Orphan ... Read more

March 31, 2021March 31, 2021

Cost of Rare Diseases in US? Nearly $1 Trillion in 2019, EveryLife Foundation Finds

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As anyone affected by a rare disease knows, treating the illness while trying to go about everyday life is an expensive undertaking. But exactly how expensive — in terms of ... Read more

March 24, 2021March 24, 2021

FAST, SFARI Partner to Launch International Research Council

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The Foundation for Angelman Syndrome Therapeutics, known as FAST, is partnering with the Simons Foundation Autism Research Initiative (SFARI) to launch the International Angelman Syndrome Research Council — or INSYNC-AS ... Read more

March 17, 2021March 17, 2021

ORCA, Reliable Measure of Communication for Angelman, in Early Use

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A reliable tool for parents or caregivers to judge the quality of nonverbal communication in Angelman syndrome patients is now available, according to the Foundation for Angelman Syndrome Therapeutics (FAST), which supported work that ... Read more

March 9, 2021March 9, 2021

Pink, Green, Blue and Purple Lights Illuminated World for Rare Disease Day

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Many diseases have their own awareness color — breast cancer is pink, muscular dystrophy is green, and AIDS is red, for example — but what’s the significance of pink, green, ... Read more

March 8, 2021March 8, 2021

The Arc Awarded Comcast NBCUniversal Grant for Special Education Initiative

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A $200,000 Comcast NBCUniversal grant has been awarded to The Arc to increase access to educational resources for students of color and economically disadvantaged youngsters with intellectual and developmental disabilities ... Read more

February 25, 2021February 25, 2021

Rare Disease Day ‘Patient Hero’ Raises Awareness Through Fashion

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Diagnosed with sickle cell disease as a 6-month-old, Tristan Lee has faced a lot of challenges over his 37 years of life. But from a young age, he also learned ... Read more

February 23, 2021February 23, 2021

Rare Disease Day at NIH, Set for March 1, Growing Year by Year

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Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place on March 1, will feature panel discussions, patient stories, research updates, TED-style talks, and a ... Read more