Ann Martin's Blog (9)

Understanding Currently Recruiting Gene Therapy Studies

For the last many months, we have all been watching gene therapy make headlines around the world, across various diseases. As you know, earlier this year PPMD launched a Gene Therapy Initiative to help support the exploration of various gene therapy technologies in Duchenne.

Of particular note has been the progress made in…


Added by Ann Martin on December 19, 2017 at 4:19pm — 1 Comment

Your DuchenneConnect Registry Data is Advancing Research! New Publication

Do you ever wonder what happens to all the data we collect in the DuchenneConnect Registry? Ever wonder why we regularly ask you to update your registry account?

How your data is used:

Your data is used in many ways.…


Added by Ann Martin on October 16, 2017 at 10:29am — No Comments

PPMD's DuchenneConnect Team Presents at Annual Genetic Conference

Last week, Ann Lucas and I had the honor of presenting an Educational Breakout Session focused on Duchenne at the National Society of Genetic Counselors (NSGC) Annual Education Conference in Seattle. Lauren Morgenroth from TRiNDS also presented during the session, as well as Laurie Paschal, a Duchenne mom living…


Added by Ann Martin on October 5, 2016 at 1:00pm — No Comments

DuchenneConnect & UCLA Recruiting Patients for Genetic Modifiers Study

We know that changes or mutations in the Dystrophin gene are what cause Duchenne and Becker but we don’t have a good understanding of why people within the same family with the same genetic change (genotype) can be so different in their symptoms (phenotype). The…


Added by Ann Martin on March 15, 2016 at 10:49am — No Comments

Update on Summit Clinical Trials

Summit Therapeutics’ SMT C1100 is a small molecule drug that targets increasing the production of utrophin. Utrophin is a protein that is present in early muscle development but “switched off” as muscle fibers mature and dystrophin is produced. SMT C1100 hopes to maintain the production of utrophin,…


Added by Ann Martin on February 9, 2016 at 1:00pm — No Comments

DuchenneConnect Annual Report: Registry Growth & Expanded Services

This past year has brought many new and exciting changes to DuchenneConnectthe robust and cutting-edge registry and resource that connects Duchenne and Becker patients with actively recruiting clinical trials and research studies, and educates patients and families about Duchenne and Becker research. The registry, which was established in 2007, has matured beyond a standard registry and into an innovative…


Added by Ann Martin on May 7, 2014 at 11:32am — No Comments

DuchenneConnect Presents 2012 Data & 2013 Update

Parent Project Muscular Dystrophy and DuchenneConnect are happy to present the 2012 DuchenneConnect Year-End Report! Read about the registry’s accomplishments in 2012 and goals for 2013. We had a busy and exciting year as we…


Added by Ann Martin on April 30, 2013 at 10:55am — No Comments

Results from DuchenneConnect Study - PPMD Webinar Follow up

Thank you for participating in last week’s webinar on DuchenneConnect data! Many of you asked questions during the webinar that we were unfortunately unable to get to within the hour. We have done our best to answer all questions submitted.

Please see the list of Q&A's following the webinar video below, and let me know if you have any additional follow up questions!…


Added by Ann Martin on May 2, 2012 at 12:00pm — No Comments

DuchenneConnect Direct Access October Webinar: Updates in Genetic Testing


The New DuchenneConnect Webinar Series allows you direct access to key figures in the fight to end Duchenne. The October webinar, Updates in Genetic Testing, is now available online! Listen here: …


Added by Ann Martin on November 3, 2011 at 8:30am — No Comments

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