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LAMA2 Community Update
August, 2020
Check out the latest updates for the LAMA2 Community
Now Recruiting:
A Retrospective Study of Natural History Data in Infants and Toddlers with LAMA2-Congenital Muscular Dystrophy
The Study
This is a non‐interventional, retrospective medical chart review study of affected individuals with LAMA2‐CMD. Medical records from approximately 100 participants born between 2000 and 2017 will be reviewed and compiled using the Congenital Muscle Disease International Registry (CMDIR).
Participation Requirements
  • Confirmed diagnosis of LAMA2‐CMD
  • Born between 2000—2017
  • Comprehensive medical records available for birth—5 years, with a special interest in birth—2 years
  • Records available in English, Spanish, French, Dutch or Arabic
  • Participants must be registered in the CMDIR
More Information
Study will close upon enrollment of 100 LAMA2 participants.

Please contact the Cure CMD Study Team for more information about this study and how to enroll.

Study Sponsors
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Meet La-Loyd the LAMA2 Llama
La-Loyd the Llama has arrived! This LAMA2-CMD community mascot is here to support and advocate for LAMA2 families, and tell the story of Merosin Deficient CMD.

La-Loyd will log his journey with CMD, engage with the affected community on social media, provide kid-friendly educational facts about LAMA2-CMD, and answer your/your child's questions.

Join us in following La-Loyd and sharing your own LAMA2 stories!

LAMA2 Global Consortium
We are pleased to share that three LAMA2 advocacy organizations are working toward a formal consortium agreement with our industry and research partners. The goal is to accelerate progress toward treatments for LAMA2-CMD, and to represent you, the affected community.

When the agreement is finalized, we will be able to share more details, but know that we are working hard on your behalf and will be in touch with more information when it becomes available.
Who is Gavin Grubs?
Check out this amazing short film featuring 19 year-old LAMA2 community member Gavin, and his family's annual ski trip to the Adaptive Sports Center in Crested Butte, Colorado.
We are thrilled to announce TWO LAMA2 research grant awards for the 2020-2022 grant cycle:

Pr. Dwi Kemaladewi, University of
Pittsburgh: Two-year grant for $50,000 to investigate therapeutic genetics and disease modeling in
LAMA2-CMD. This study will use fibroblast cells donated from affected individuals to fully explore the potential of CRISPR technology for use in treatments.

Pr. Francesco Muntoni, University College London: 18-month grant for $50,000 to investigate in vivo modulation of modifier genes for LAMA2-CMD. This follow-up study will take results from this team's 2018-2020 funded project to further expand our understanding of the role that other genes may play in an affected individual's symptoms and severity.

These grants could not have been funded without you, our community of dedicated supporters. Special thanks to S.A.M. (Struggle Against Muscular Dystrophy) for making a second LAMA2 grant possible!

Learn more about our research strategy and funding priorities on the Cure CMD website, and check out the latest research papers in our Publication Library.
After 11 Years, the Congenital Muscle Disease International Registry (CMDIR) is Getting an Upgrade.

We are almost ready to launch a significant platform upgrade to enhance the way your, or your child’s, data is tracked and reported on.

The registry has grown to more than 3,000 affected individuals from 86 countries, and it's time for a face lift! The new registry will offer:

  • A mobile-friendly interface
  • Integrated educational materials relevant to you
  • Easy data-sharing opt-ins
  • Intuitive analytics
  • Full-site, multi-language support, launching in English and Spanish, and expanding to additional languages over the next two years
  • Flexible, comprehensive data reporting useful to researchers and industry working to advance the clinical trial pipeline.

Already registered? Look for an email soon inviting you to create your new CMDIR login.

Haven't registered yet? Now is the time! We need you to make treatments for LAMA2-CMD possible!
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Connecting with the CMD Community has never been easier!