The Results are in! Cure CMD’s First LA Marathon a Great Boon for CMD Research

The second weekend of March brought the CMD community together in a brand-new way this year with the Los Angeles Marathon, when more than 200 donors helped raise more than $21,000 for CMD research. A total of twenty-seven runners participated in the 2018 LA Marathon – eight team members participated in the the LA Big 5k, twelve ran the 13.1 mile Charity Challenge, and seven team members ran the full marathon, a whopping 26.2 miles. We are so proud of Team Cure CMD, who spent months training, raising funds, planning their travel, and sharing with their family and friends all about Cure CMD’s mission to support research toward treatments and a cure for CMD. The entire L.A. Marathon effort reac

Gene Correction Therapy Study, Seed-Funded by Cure CMD, Continues through MDA Grant

What: A $180,000 grant has just been announced by the Muscular Dystrophy Association (MDA) to fund the exploration of potential of gene correction therapy in merosin-deficient congenital muscular dystrophy (MDC1A), which is caused by mutations in the LAMA2 gene. If successful, this work could provide the necessary preclinical data and comprehensive strategy to bring CRISPR/Cas9-mediated gene correction closer to clinical application. For several years, Cure CMD seeded this project with a $100,000 grant award to the Cohn Lab, in addition to providing support for the improvement of the LAMA2 mouse model, critical to this research. Funding of early research such as this is what sets Cure CMD a

It's Volunteer Week and here's our next highlight: Susan Lee-Miller

Susan is an "OG" mom who has a son with COL6-CMD. Susan has supported Cure CMD's mission from Day 1. Her ongoing engagement in fundraisers and community outreach, and as an experienced CMD mom is an incredible asset. Some of Susan's contributions include: Co-coordinator for the Million Dollar Bike Ride, an annual event sponsored by UPENN to raise funds for research grants in CMD (see our blog post about this year's event here to find out how you can support the team Moderator of our first support group on Yahoo (2008!) for COL6-CMD CMD Community Liaison, reaching out to CMD affected individuals that appear in news feeds to connect them to our community. CMD Traveling Ambassador, through supp

It's Volunteer Week and here's our next highlight: Diane Smith-Hoban

Diane is mother to two wonderful young men, one of whom has COL6-CMD. Diane is one of our "OG" moms - a Cure CMD supporter even before Cure CMD was officially formed. Since 2008, Diane has made progressing our mission a priority, contributing to numerous projects and fundraisers including: Co-coordinator for the Million Dollar Bike Ride, an annual event sponsored by UPENN to raise funds for research grants in CMD (see our blog post about this year's event here to find out how you can support the team) Primary author for the CMD Family Guide, a vital publication for families to learn how best to manage care for CMD. Diane also managed the translation of this guide into more than 10 languages.

It's Volunteer Week and Here's Our Next Highlight: Robin Swallow

Robin's two grandsons were diagnosed with SEPN1-RM in late 2014 after a terrible diagnostic odyssey that led them through three states and countless doctors. Soon after, Robin started researching organizations that support CMD, and came across Cure CMD, whose offices were located literally 10 minutes from her home! And so, since 2015, Robin has been our "boots on the ground" local volunteer, ensuring our office continues to run smoothly and efficiently. Robin not only dedicates several hours a week in the office (earning her own desk!), she also puts in countless hours from home. Some days, it's 4am and Robin's emailing, updating the registry, editing files. Some of her past and ongoing ta

2018 Tax Law: What We Know for Sure

Cure CMD and the impact of the new tax law As many of you know, there is something about living with a rare condition that gives affected individuals, their families and caregivers the ability to adapt to change and handle uncertainty on a daily basis, year in and year out. Cure CMD mirrors this trait and has taken a moment to reflect on the impact of the new tax law on our work and has found ways to...adapt. What we know for sure is that our community of supporters gives first because of a commitment to the belief that together – a cure is among us. ​ Just the Facts First, a cliff note version of the tax plan according to a recent article from the New York Times - "The tax plan approved by

It's Volunteer Week and Here's Our First Highlight: Charlene York

Char has been a mainstay of Cure CMD almost since inception. Mom, wife, sister-in-law and daughter-in-law to four (and counting) CMD-affected family members, Char has dedicated her (early) retirement to our cause. The number of projects over the past 10 years that Char has been instrumental in completing cannot be fully catalogued, but here are some highlights: A constant and insistent voice for CMD at state and federal rare disease events Management of the CMD Scientific Publication Library Management of the CMD Researcher Database Travel & hotel for over 150 and hotel contract negotiation for 2017 CMD SciFam Travel & hotel for more than 80 attendees to our four 2016-2017 CMD Scientific Mee

Santhera Announces Successful Completion of First Clinical Trial with Omigapil in Patients with Cong

Please see the end of this article for Frequently Asked Questions - CALLISTO Phase 1 Clinical Trial Pratteln, Switzerland, April 5, 2018 - Santhera Pharmaceuticals (SIX: SANN) reports the successful completion of the first clinical trial with omigapil in patients with two forms of congenital muscular dystrophy (CMD) conducted in the US at the National Institutes of Health (NIH). The ascending multiple dose cohort study (CALLISTO) met its primary objective to establish a favorable pharmacokinetic profile of omigapil and demonstrated that the study drug was safe and well tolerated in children and adolescents with CMD. Following further data analysis, the Company will discuss these results with

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