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Edasalonexent Slows DMD Progression in Phase 2 Trial

Phase 2 study results of edasalonexent, an investigational oral small molecule NF-kB inhibitor for the treatment of Duchenne muscular dystrophy (DMD), suggest that the therapy is associated with slowed disease progression, as well as a good safety profile over the course of more than 50 patient-years of exposure in boys with the disease.1 Treatment with […]


Why Canada needs a comprehensive rare disease strategy

With over 7,000 rare conditions already identified and dozens more being discovered every day, it is estimated that rare diseases impact 1 in 12 Canadians, two-thirds of whom are children. But each disease affects only a handful of individuals, so understanding and medical expertise are limited and uneven across the country. Many of these conditions […]


PTC Therapeutics Announces First-Ever Winners of Its PRIORITY Program To Support Research Projects In Rare, Genetic Disorders

– $545,000 awarded to help improve Duchenne muscular dystrophy diagnosis and screening across the globe –SOUTH PLAINFIELD, N.J., May 20, 2019 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced the three funding recipients from PRIORITY, an annual program to support outstanding clinical research by the rare genetic disorders community. This year’s funding will accelerate innovative research projects to improve Duchenne […]


Helping Canadians get faster access to the prescription drugs they need to live healthier lives

Government of Canada consults on new approach to fast-track the review of treatments for serious, rare and debilitating diseases OTTAWA, May 13, 2019 /CNW/ – The Government of Canada is committed to helping all Canadians access affordable treatment options they need to help them improve and maintain their health. For many Canadians, managing the symptoms of rare or debilitating diseases can be […]


EGF Protein Preserves Muscle Strength, Promotes Muscle Regeneration in DMD Mice, Study Shows

A protein called epidermal growth factor (EGF) can help preserve muscle strength and increase muscle regeneration in a mouse model of Duchenne muscular dystrophy (DMD), a finding that may pave the way for new treatment strategies for DMD, researchers said. The study, “EGFR-Aurka Signaling Rescues Polarity and Regeneration Defects in Dystrophin-Deficient Muscle Stem Cells by Increasing Asymmetric Divisions,” […]


Translarna™ (ataluren) is the First Therapy Approved in Brazil for Duchenne Muscular Dystrophy

– Ambulatory Duchenne patients who are 5 years and older with a nonsense mutation can now access a treatment that targets the underlying cause – SOUTH PLAINFIELD, N.J., April 29, 2019 /PRNewswire/ — PTC Therapeutics, Inc. today announced that Translarna™ (ataluren) has been granted marketing approval from the Brazilian National Health Surveillance Agency (ANVISA) under rare diseases […]


Raising Both Dystrophin and Utrophin Levels May Rescue Muscles in DMD, Mouse Study Suggests

Using a genetic engineering tool to restore dystrophin while raising levels of utrophin, a similar protein, leads to better improvements in muscle function than either approach alone, research in a mouse model of Duchenne muscular dystrophy (DMD) reports. The study, “The potential of utrophin and dystrophin combination therapies for Duchenne muscular dystrophy,” appeared in the journal Human Molecular […]


Treating Duchenne muscular dystrophy focus of new research partnership

Toronto, Ontario – Muscular Dystrophy Canada (MDC) and The Foundation for Gene & Cell Therapy (Jesse’s Journey) are joining forces to accelerate ground-breaking research focused on new treatments for Duchenne muscular dystrophy to the sum of $600,000. Through this partnership, MDC will provide $300,000 with Jesse’s Journey matching the commitment. Two research projects will be […]


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