The International Society for Mannosidosis and Related Diseases (ISMRD) and the National MPS Society are joining forces to urgently raise critical funding to advance groundbreaking gene therapy research for Mucolipidosis II, ll/III, and lll at Washington University in St. Louis.
Mucolipidosis II, ll/lll, and III are devastating, progressive, and life-threatening lysosomal diseases that impact nearly every aspect of a person’s life. Children and adults living with Mucolipidosis face significant physical challenges, chronic pain, loss of mobility, respiratory complications, cardiac involvement, and reduced life expectancy. Currently, there are no treatment options, and care is largely supportive rather than curative.
This research represents more than scientific progress, it represents hope for families who have spent years waiting for meaningful therapeutic advancements. Gene therapy has the potential to fundamentally change the future of this disease, but continued progress depends on immediate and sustained funding support.
Time is critical. Every day without treatment advancement is another day families face the devastating progression of this disease. Every delay places lives at risk and prolongs the urgent need for effective therapies.
Your contribution goes directly toward supporting this important initiative and helping move this research forward at a pivotal stage of development. With your support, we can help accelerate progress toward transformative therapies for individuals and families affected by Mucolipidosis.
Thank you for your generosity, advocacy, and commitment to this mission. Together, we can help bring hope to a community that urgently needs it.
The Nonprofits Behind the Stride
Proceeds from this initiative, raised through both ISMRD and the National MPS Society, will directly support the development of the gene therapy program at Washington University in St. Louis, advancing critical research efforts toward a potential treatment for Mucolipidosis.
Please designate your donation to the ML Challenge Grant
ISMRD was incorporated as the International Society for Mannosidosis & Related Diseases on March 10, 1999. Founded by Paul and Debora Murphy, parents of a child diagnosed with Alpha-Mannosidosis. While inspired by their family’s experience with Alpha-Mannosidosis, ISMRD was established to support the glycoproteinoses community. a subset of ultra-rare lysosomal diseases that historically lacked coordinated advocacy, family support, and research collaboration.
ISMRD not only serves affected individuals and families, but also scientists, researchers, and physicians dedicated to advancing research, improving care, and exploring future treatment options for these disorders worldwide.
ISMRD continues to seek a future in which children with Glycoprotein Diseases can be detected early, treated effectively, and go on to live long, healthy and productive lives.
ISMRD was incorporated as the International Society for Mannosidosis & Related Diseases on March 10, 1999. Founded by Paul and Debora Murphy, parents of a child diagnosed with Alpha-Mannosidosis. While inspired by their family’s experience with Alpha-Mannosidosis, ISMRD was established to support the glycoproteinoses community. a subset of ultra-rare lysosomal diseases that historically lacked coordinated advocacy, family support, and research collaboration.
ISMRD not only serves affected individuals and families, but also scientists, researchers, and physicians dedicated to advancing research, improving care, and exploring future treatment options for these disorders worldwide.
ISMRD continues to seek a future in which children with Glycoprotein Diseases can be detected early, treated effectively, and go on to live long, healthy and productive lives.
The mission of the National MPS Society is to support individuals and families affected by mucopolysaccharidoses (MPS), mucolipidosis (ML), and related diseases through advocacy, research, education, and support services.
The organization works to advance scientific and medical research, improve access to care and treatments, raise public awareness, and provide resources and community connections for patients and caregivers navigating these rare disorders.