About Cure CMT4J
Cure CMT4J was formed as the Talia Duff Foundation in Spring 2016 to raise funds to support the research, development, and production of a therapy or cure for Charcot Marie Tooth Disease Type 4J. The inspiration for the foundation is Talia Duff (her story below) and the hope for a treatment for not only Talia’s rare disease, but to advance gene therapy as treatment for any number of rare, genetic diseases.
Cure CMT4J / The Talia Duff Foundation Inc. (Tax ID: 81-3019217) is a Massachusetts corporation. Federal tax exempt status as a public charity under Section 501(c)(3) has been approved by the U.S. Internal Revenue Service.
About Talia Duff
At just ten years old, Talia is known to many in her community. She is the bright light among fifth graders who love to encircle her at the lunch table or play her imaginative games at recess. She is a big reader, a budding musician, actor, tandem cyclist and sit-skier. Talia is the center of a very special family and community who help her to embrace all that life has to offer. She is also brave beyond her years, having endured countless medical procedures and interventions for most of her life.
After years of failed treatments, surgeries and unanswered questions, Talia’s family recently learned that she has an ultra-rare, genetic disease known as Charcot Marie Tooth Type 4J (CMT4J). This would finally explain why she has never been able to walk independently or run or jump with her friends. The disease is progressing rapidly. In less than a year CMT4J has taken Talia from walker to wheelchair, robbed her of strength in her arms, and weakened the nerves involved with breathing.
After recent trips to meet with specialists in CMT4J Talia’s family learned that there is Hope. Gene therapy offers great promise for a treatment or cure. Diseases such as CMT4J that are mono-genic (single-gene defects) are the most ideal candidates for gene therapy. Successes in gene therapy have been seen in hereditary blindness, immune deficiencies, such as “the bubble baby”, hemophilia, lysosomal storage diseases and leukemia. Currently there are over 600 clinical trials on-going using gene therapy. Unfortunately, ultra-rare diseases like Talia’s receive little attention or funding. Other parent- or patient-driven models of taking on the task of fundraising and expediting the science through to clinical trials at a faster rate show great promise.
Who’s Who at Cure CMT4J
Among the many friends and family members helping to drive this effort forward are:
Doug Deangelis – Development
Jocelyn Duff — Co-founder
John Duff – Co-founder
Bill Gorman – Scientific/Medical Advising
Marcia Gray – Communications and Media Relations
Jeff Jones – Operations Management
Frank Hertz – Website and Email
Gretchen Marinopolous – Fundraising
Toni Mooradd – Fundraising
Shane Murray – Scientific/Medical Advising
Michele Wertz – Development