Happy New Year!

News and Updates

Feb 2023

Please Join our Upcoming Webinar in the Community Conversations series!

We invite all who are interested to join the upcoming webinar on Saturday, February 11th, that we are hosting to provide the community an opportunity to hear an update directly from Astellas Gene Therapies.

Click here to Register

Clinical Trials and the Patient Voice

When research is published in medical journals, the focus is usually on technical aspects such as patient characteristics, quantifiable symptoms, and outcomes or side effects. But the real-life experience of the participants or potential participants is largely missing from the scientific literature so far. MTM-CNM Family Connection is deeply grateful for the recent opportunity to collaborate with Dr. Nicol Voermans and her team at Radboud UMC (Netherlands), ZNM Zussamen Stark, and the Myotubular Trust on a research project to collect and analyze patient and family feedback on the preclinical study and clinical trial experience. Several of our community members in the US joined a confidential focus group discussion to share their perspectives. Their insights will be added to those from three similar focus groups that took place in Germany and England, and will provide valuable lessons that can influence the conduct and design of future studies and clinical trials. We are eager to hear their analyses and will update the community when the findings are published! Thank you to all who participated and are working to amplify the voice of the people most affected by clinical trials--the patients and their families.

Special Thank you to the Griese Family!

On November 5, 2022, the Griese Family of TX, led their team, "Zekie's Posse," to honor their sweet, little boy Ezequiel, in the El Paso Flying Horse Half Marathon, 10k & 5k presented by the City of Sunland Park.

We're thankful that, through the Griese family efforts, MTM-CNM Family Connection was one of the selected charities for the race!

Thank you, Lorena, Thomas, Ezequiel, and all your friends and loved ones who joined in and raised awareness and support for our amazing community!

Watch the video for Zekie's Posse!

Our Community Impacting the World!

Oluronke Kalejaiye, the matriarch of one of our beautiful MTM families, recently shared with us how much the conference and community connections and resources have meant to her, her spouse Kelvin and their three adorable children, especially their son Prince, who has MTM. While overcoming challenges and fighting the frequent flaws in the US health system here on behalf of their son, the Kalejaiye family has also held close in their hearts the people of her home country Nigeria, where there are even fewer resources and accommodations for those needing complex medical care.

This love and compassion for the people of their native land has inspired Oluronke and Kelvin to launch a new foundation, Treasured Kids, that will focus on spreading awareness and understanding of special medical conditions in Nigeria. Their mission is to break down some of the barriers and biases that hinder medically-complex children and their families from being truly accepted and fully embraced by their local community.

The Treasured Kids Foundation will provide awareness, education, advocacy, and support to families, communities, and the Nigerian society at large. The Kalejaiyes said they are starting out small for now, but just like a seed about to flourish, we believe many great things will come in the future for the work the Kalejaiye family is initiating and are so excited to see what blessings are in store! We invite you to follow their efforts at Treasured Kids Foundation at these social media links:

Twitter: @TreasuredkidsNG

Instagram: treasuredkidsng

Facebook: TreasuredKidsFoundation

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