Accelerate research and treatment for LGMD R5/2C
Accelerate research and treatment for LGMD R5/2C
Le problème
No parent should ever have to hear the devastating words, "There is no treatment, no cure. Go home and enjoy the time while your child is still walking." These were the haunting words my close friend heard when his 22-month-old son, Samy, was diagnosed with Limb-Girdle Muscular Dystrophy Type 2C/R5 (LGMD R5/2C). This diagnosis forever changed their family’s lives, capturing them in a world of uncertainty and despair.
Samy's father is one of my closest friends, and watching him and his family navigate the heart-wrenching reality of watching their precious child lose physical mobility is painful beyond words. This is an ultra-rare genetic disorder, and the lack of accessible treatment options and clinical trials makes the battle even more daunting. Despite these overwhelming challenges, their unwavering determination to advocate for improvements through the Hope for Samy Foundation is a beacon of hope.
Limb-Girdle Muscular Dystrophy Type 2C/R5 is a rare genetic disorder that causes progressive muscle weakness and wasting, primarily affecting the hip and shoulder regions. The absence of local clinical trials and the lengthy process of gaining access to emerging gene therapies is suffocating for families desperately seeking a lifeline for their children. It is imperative to improve the pace at which treatments are developed and made available to those affected by this cruel disease.
I am calling on healthcare authorities, research institutions, and policymakers to expedite research and clinical trials for LGMD R5/2C. We need increased funding for gene therapy research and international collaboration to bring hope to families like Samy’s. Early and accessible treatments can dramatically improve the quality of life for these children and give families the comfort of knowing they’ve done everything possible.
Join me in urging the medical community and our leaders to focus on accelerating the path to treatment for LGMD R5/2C. Samy and countless other children deserve a fighting chance. Please sign this petition to support our cause and make a tangible difference in the lives of families grappling with this challenging condition.
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Le problème
No parent should ever have to hear the devastating words, "There is no treatment, no cure. Go home and enjoy the time while your child is still walking." These were the haunting words my close friend heard when his 22-month-old son, Samy, was diagnosed with Limb-Girdle Muscular Dystrophy Type 2C/R5 (LGMD R5/2C). This diagnosis forever changed their family’s lives, capturing them in a world of uncertainty and despair.
Samy's father is one of my closest friends, and watching him and his family navigate the heart-wrenching reality of watching their precious child lose physical mobility is painful beyond words. This is an ultra-rare genetic disorder, and the lack of accessible treatment options and clinical trials makes the battle even more daunting. Despite these overwhelming challenges, their unwavering determination to advocate for improvements through the Hope for Samy Foundation is a beacon of hope.
Limb-Girdle Muscular Dystrophy Type 2C/R5 is a rare genetic disorder that causes progressive muscle weakness and wasting, primarily affecting the hip and shoulder regions. The absence of local clinical trials and the lengthy process of gaining access to emerging gene therapies is suffocating for families desperately seeking a lifeline for their children. It is imperative to improve the pace at which treatments are developed and made available to those affected by this cruel disease.
I am calling on healthcare authorities, research institutions, and policymakers to expedite research and clinical trials for LGMD R5/2C. We need increased funding for gene therapy research and international collaboration to bring hope to families like Samy’s. Early and accessible treatments can dramatically improve the quality of life for these children and give families the comfort of knowing they’ve done everything possible.
Join me in urging the medical community and our leaders to focus on accelerating the path to treatment for LGMD R5/2C. Samy and countless other children deserve a fighting chance. Please sign this petition to support our cause and make a tangible difference in the lives of families grappling with this challenging condition.
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Pétition lancée le 28 juillet 2026