Dynacure Recognizes Rare Disease Day 2021

STRASBOURG, France and PHILADELPHIA, Feb. 26, 2021 /PRNewswire/ -- Dynacure, a clinical-stage company focused on developing and commercializing novel therapies to transform the lives of patients with rare diseases who have limited or no treatment options, today announced its recognition of Rare Disease Day 2021. Dynacure is helping to raise awareness of myotubular and centronuclear myopathies, or CNM, by publishing a series of stories from patients and families who have been affected by this group of rare and life-threatening disorders.

These CNM family stories can be accessed on Dynacure's website and feature families in both the United States and in Europe. Families discuss the often-complicated and delayed path to an accurate diagnosis, the struggles of caring for medically fragile young patients and the increased challenges brought by the COVID-19 global pandemic. Families also discuss finding joy in the simple things in life, the value of being connected to a broader CNM community and their hopes for the future.

"An important aspect of our work with the Myotubular and Centronuclear Myopathy community is to connect affected families with each other, and in doing so, we realize that we are not alone, we have much in common and that we are stronger together," said Dr. Holger Fischer, Founder and President of the ZNM - Zusammen Stark! e.V., a patient organization in Germany committed to supporting those with CNM, and added "It is important that we keep telling our stories as a way to raise awareness, to support each other and to celebrate the lives of those affected by CNM. These are our goals on Rare Disease Day and always."

"There is no better way to understand and appreciate any rare disease than through the stories of the patients, families and caregivers who are affected," said Dynacure Chief Executive Officer Stephane van Rooijen. "Rare Disease Day is an opportunity to listen to the stories from the rare disease community, and as an industry to acknowledge that there is great work ahead of us and potential to make a difference. I want to thank those in the myotubular and centronuclear myopathy community who have taken the time to share their stories with us. We hear you – message understood."

Myotubular and Centronuclear Myopathies are serious, rare, life-threatening disorders that affect skeletal muscles from birth. People with CNM begin experiencing muscle weakness at any time from birth to early adulthood and many patients die within the first 18 months of life. Patients who survive longer require intense medical management and nearly uninterrupted support, including permanent ventilation, brace with head support and feeding tubes.  Myotubular and Centronuclear Myopathies affect an estimated 4,000 to 5,000 patients in the European Union, United States, Japan and Australia ¹.

Rare Disease Day (www.rarediseaseday.org) was established by EURORDIS in 2008 and is held on the last day of February each year in an effort to build awareness of rare diseases and the impact they have on patients and their families. The theme for Rare Disease Day 2021 highlights the need to provide members of the rare disease community around the world with new opportunities to meet, share insights and support one another, and join together in efforts to educate and advocate. This year will mark the first all-digital Rare Disease Day involving interactive online events planned by hundreds of international advocacy, research and patient care organizations. Please visit Dynacure's website to read the CNM family stories.

About Dynacure

Dynacure is a clinical-stage company focused on developing and commercializing novel therapies to transform the lives of patients with rare diseases who have limited or no treatment options. The Dynacure team leverages its proven track record in rare disease drug development to build a pipeline of novel drug candidates. Dynacure is developing DYN101, an investigational antisense product candidate designed to reduce the expression of dynamin 2 protein for the treatment of Myotubular and Centronuclear Myopathies, in strategic collaboration with Ionis Pharmaceuticals. Dynacure is also building a complementary research portfolio targeting other orphan disorders, including its DYN201 program for the treatment of Hereditary Spastic Paraplegias (caused by mutations in the SPG11 gene).

Dynacure is headquartered in Strasbourg, France with a corporate office in Philadelphia, PA, USA.

For more information, please visit www.dynacure.com.

1. Neuromuscul Disord. 2018 Sep;28(9):766-777. doi: 10.1016/j.nmd.2018.06.012. Epub 2018 Jul
2. Nat Commun. 2017 Jun 7;8:15661. doi: 10.1038/ncomms15661.
3. Proc Natl Acad Sci U S A. 2018 Oct 23;115(43):11066-11071. doi: 10.1073/pnas.1808170115. Epub 2018 Oct
4. (Cowling et al 2014 JCI)

SOURCE Dynacure

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