Category Archives: Cure CMD News

Cure CMD News

Standard Operating Protocols to Evaluate CMD Mouse Models

Cure CMD has been working with the scientific community to develop standard operating protocols to evaluate CMD mouse models.  This work has been co-funded by the AFM and led by Dr. Raffaella Willmann. Developing a standard way to evaluate mouse models, assists Cure CMD in evaluating the size of the effect of any intervention and […]

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LMNA (L-CMD) Fundraing Campaign to Support Ongoing Research

Cure CMD has supported research for a number of projects – to date funding over $1.5M in research grants. Currently, Cure CMD is funding two of the world’s leading scientists in LMNA research: Drs. Gisele Bonne and Antoine Muchir. The Dague Family has established a fundraising campaign to help ensure this valuable research continues to […]

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Using Patient Registries and Multi-Gene Panels: A Paradigm Shift in the Pursuit of Genetic Diagnosis for Rare Pediatric Disorders

Patient registries provide a powerful resource to identify undiagnosed patient cohorts and collect cross-sectional and longitudinal clinical information on those individuals. For rare disorders, such as the congenital myopathies, a patient registry with curated patient-reported and medical report data may serve as the ideal tool to focus molecular testing. Indeed, molecular testing, which utilizes next […]

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Diagnostic Approach to the Congenital Muscular Dystrophies

Abstract Congenital muscular dystrophies (CMDs) are early onset disorders of muscle with histological features suggesting a dystrophic process. The congenital muscular dystrophies as a group encompass great clinical and genetic heterogeneity so that achieving an accurate genetic diagnosis has become increasingly challenging, even in the age of next generation sequencing. In this document we review […]

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Abbey & Izzie Etsy Shop Donates 10% of March Proceeds to Cure CMD

10% of the proceeds from the Abbey & Izzie Etsy shop will go to Cure CMD during the month of March! They make handmade party favors. Click here to shop!

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Winter 2014 Nemaline Myopathy Newsletter

Click here to view the Winter 2014 Nemaline Myopathy Newsletter

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Prothelia and the University of Nevada Enter Into Exclusive Strategic Agreements with Alexion for MDC1A

Experimental Protein Replacement Therapy for Congenital Muscular Dystrophy Type 1A (MDC1A) MILFORD, Mass. and RENO, Nev., Feb. 13, 2014 /PRNewswire/ — Prothelia Incorporated (Prothelia) and University of Nevada, Reno announce that they have entered into strategic agreements with Alexion for the development of Laminin-111, a patented experimental protein replacement therapy for merosin-deficient congenital muscular dystrophy […]

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Earn Donations for Cure CMD Through Survey Monkey

Cure CMD is partnering with SurveyMonkey Contribute! Take opinion surveys on SurveyMonkey Contribute, and they will donate $0.50 for every survey you answer. Click here to sign up: 

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Cure CMD Funds 3 New Research Grants in 2014

Losartan Trial in Dyw Mice- Dr. Sweta Girgenrath. This drug trial in the LAMA2-CMD mouse model will look at whether early treatment (two weeks) and late treatment (4 weeks) confers the same benefit and what the benefit of sustained treatment is.  This is a two year grant that will evaluate biomarkers in mouse muscle and […]

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Kansas City Marathon Runners Raise $132,000 for Cure CMD

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  • Congenital Muscular Dystrophy

    A group of diseases causing muscle weakness at birth. Several defined genetic mutations cause muscles to break down faster than they can repair or grow. A child with CMD may have various neurological or physical impairments. Some children never gain the ability to walk, while others lose the ability as they grow older. Learn more...

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