Dec. 1, 2015
TOPIC: Featured News
Posted by Lisa Sencen
The Joshua Frase Foundation (JFF) is a non-profit organization whose mission is to support ongoing medical research in hopes of finding a cure for myotubular myopathy. JFF also provides monetary and emotional support to families who are affected by congenital myopathies. Myotubular myopathy is a rare muscle-wasting disorder that occurs in three forms based on severity, inheritance, and symptoms. X-linked myotubular myopathy, the most severe form, is generally present at birth or occurs in infancy. Autosomal recessive myotubular myopathy is a less severe form that usually occurs during infancy or childhood. The least severe form, autosomal dominant myotubular myopathy, usually presents between the first and third decades of life and is slowly progressive.
1.) What does it mean to you personally to be a patient organization serving the rare community?
It is an honor to be able to reach our families and give them the hope and the support they need on the journey of rare disease. No parent should walk this journey alone. It takes a village to care for these critically ill children.
2.) What do you find your patient community values most from your organization?
First and foremost, when new family’s are being given death sentences on the survival of their infants, the foundations resources and educational material are an asset that gives hope in an otherwise hopeless situation.
3.) What are some of the challenges your organization has faced?
Consistent loss of these precious children, even our own while we anxiously await a therapy or cure. We are also challenged by the lack of knowledge on the care guidelines for these affected children by medical professionals.
4.) Whats been your most successful awareness campaign and/or fundraising event?
For 12 years we rode the wave of Wall Street, the NFL, and the music industry and raised over 6.2 million between 1997 and 2009.
P.O. Box 2041
Ponte Vedra Beach, FL 32004 USA
NORD is happy to be putting the spotlight on the Joshua Frase Foundation this week. You can look forward to social media posts on our Facebook and Twitter accounts about JFF throughout the week! Member Organizations to NORD interested in Member Spotlight should contact their regional membership managers at NORD to learn more!