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 13, 2021April 13, 2021

First Patient Dosed in FORTIS Trial of AT845 for LOPD

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The first patient has been dosed in the FORTIS Phase 1/2 trial of AT845, a single-dose gene therapy for adults with late-onset Pompe disease (LOPD). AT845 is designed to deliver a ... Read more

April 6, 2021April 6, 2021

Upcoming International Pompe Day Aims to Raise Awareness

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In an effort to raise public awareness and increase understanding of Pompe disease, the 8th Annual International Pompe Day will be celebrated on April 15. This year’s theme is “Pompe ... 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 30, 2021March 30, 2021

Maze Therapeutics Aims to Bring Oral Pompe Therapy Into Clinical Testing

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Maze Therapeutics announced its first three lead therapeutic candidates, including an investigational oral medication designed to treat Pompe disease. Pompe disease is caused by genetic mutations that make the body unable ... Read more

March 23, 2021March 23, 2021

Gene Therapy Trial Recruiting Adults with Late-onset Pompe in US

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A Phase 1/2 trial is recruiting adults in the U.S. who have late-onset Pompe disease to test a gene therapy, AT845, being developed by Audentes Therapeutics, according to an update ... Read more

March 16, 2021March 16, 2021

#MDA2021 — Miglustat’s Role in AT-GAA as Possible Therapy Described

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Miglustat — a component of AT-GAA, an investigational therapy for late-onset Pompe disease (PD) — enhances the activity of the other component in AT-GAA, cipaglucosidase alfa, researchers report. These findings were ... Read more

March 10, 2021March 10, 2021

Experts Warn Against Interrupting Pompe Treatment During Pandemic

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At a recent webinar, experts cautioned that it could be dangerous for people with Pompe disease to stop treatment due to the COVID-19 pandemic. The disease that would come to ... Read more

March 5, 2021March 5, 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 3, 2021March 3, 2021

Myozyme Effectiveness in LOPD Fades Over Time, Real-world Study Finds

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In the first years of treatment, Myozyme (alglucosidase alfa) — marketed in the U.S. as Lumizyme — can effectively increase physical endurance, and improve lung function and muscle strength in adults ... Read more