Conference gives families hope
My husband Harold and I were fortunate to attend the Mucolipidosis Type 4 (ML4) Foundation Family & Research Conference last month in Atlanta. ML4, a relatively unknown genetic disorder, is one of 60 lysosomal disorders that affects the brain, cells, eyes, digestive system, kidneys and an ability to walk, talk and function. It
also has similarities to other diseases such as Alzheimer’s, Parkinson’s and Muscular Dystrophy.
In attendance were 14 families and their children who are afflicted with the ML4 genetic disorder who came from Australia, Israel, Brazil and nine different cities in the United States. Twenty-one scientists and researchers traveled from Israel, Wales and eight U.S. states.
For almost 30 years ML4 has been considered a Jewish genetic disorder. This is no longer the case. Of the 14 families who attended this conference, six were non-Jewish couples and one was an interfaith couple.
We attended this conference because our grandson Ryan Gale, son of Jon and Sandy Gale of Kansas City, has ML4. The conference gave us, as grandparents, the opportunity to spend three incredible days sharing experiences and hope with other families who live with this disease. It was heartwarming and heart breaking.
Bringing these families and researchers together provided us much education about the disease, including where research is going and where it needs to go. The conference provided a safe place to share our fears, tears, hardships and joys. The children are beautiful even with their disabilities, as is our own grandson. Their parents were loving and patient. I found it very difficult to leave my new family and the hard-working and compassionate researchers.
The executive director of the MLF Foundation has encouraged researchers to consider collaborating. This helps research move more quickly and prevents repetition of work being done at other labs. With government and state funding disappearing due to the economy, collaboration is one way researchers — who are both medical doctors and scholars — can continue to try to find a cure for the disease. While at the conference we embraced the willingness of the scientific presenters to collaborate and share their research with the families and other scientists.
This is a cause very near and dear to our hearts. You can learn more about ML4 by watching Adina Katz’s award-winning documentary “ML4:A Rare and Devastating Disease,” on YouTube, https://www.youtube.com/watch?v=YkpCc7SbPs8. It is a beautiful documentary about ML4 families and their children.
We would also love to hear from anyone who has questions about the research currently being conducted to find a cure for ML4, wants to learn about genetic testing or would like to make a donation toward research. I can be reached 913-402-8718.
Janet Price
Leawood, Kan.