The heartwarming story of students from three Massachusetts high schools banding together to help raise money to find a cure for the extremely rare neuropathy Charcot Marie Tooth, Type 4J (CMT4J) was featured in the Boston Globe recently.
Students from Hamilton-Wenham, Ipswich, and Danvers high schools launched a Twitter campaign to help the Cure CMT4J foundation, which was started by the parents of Talia Duff, a 12-year-old girl from Ipswich, Mass., who was diagnosed with the rare disease in 2015. Talia already had Down syndrome and the CMT4J diagnosis came with the devastating news that there was no cure.
With only 20 people estimated to have CMT4J worldwide, there was virtually no investment in research to find a cure. Talia’s parents refused to give up, and started investigating what they could do.
“We went online and started doing whatever research we could,” John Duff, Talia’s father, said in the Boston Globe news story written by Laura Elyse King.
The Duffs started the Talia Duff Foundation, now called Cure CMT4J. Their goal was to raise money for a cure. They eventually met Dr. Jun Li, at Vanderbilt University, with whom they partnered to seek a potential cure for the rare condition through gene therapy.
In mice studies, he had developed a treatment that had successfully stopped disease progression and even reversed some of the damage.
But in order to now take this research to the next step, Cure CMT4J needs to raise $1 million by the end of 2017 so the potential treatment can advance to clinical trials in humans in 2018, provided the U.S. Food and Drug Administration (FDA) grants approval.
To achieve this goal, the Foundation has been fundraising non-stop since the beginning of the year. They were relatively successful in spreading the word, but it was only after Talia’s school community thought of using Twitter as a platform to raise money and awareness that Cure CMT4J crossed the 60 percent line toward its goal.
Math teacher Kathleen Simms put together a small business project with her students to sell gift-ready jars of holiday cookie dough ingredients, which they then decorated with Cure CMT4J ribbons. And business teacher Laura Wheeler brainstormed with her students until they came up with the idea to do a “twitterstorm” – sending celebrities their message, using the hashtags #WeNeedAnAngelDonor and #RUDuffEnough and linking to a video of Talia.
The Ipswich Middle School students mostly tweeted celebrities like Ellen DeGeneres and Jimmy Kimmel. A total of 12,000 tweets were sent and they got the attention of the New England Patriots, who tweeted a link to the video that same night, challenging all #PatriotsNation to donate to the cause. The story also made it to ABC World News Tonight, where David Muir shared the plea using the hashtag #AmericaStrong.
To help Cure CMT4J reach its goal before the end of 2017, you can donate on their website and/or share Talia’s story through your own social media channels, like Twitter, Facebook, or Instagram, or by word of mouth, by telling your friends, neighbors or coworkers about the campaign. The Foundation welcomes all efforts.
CMT4J is an autosomal recessive disease caused by a mutation on the FIG4 gene, located on chromosome 6. Typically, the parents of a CMT4J patient are both carriers of the mutation. People with CMT4J can experience progressive muscle weakness, difficulty walking, or wheelchair dependence. They can lose the ability to use their arms or suffer from joint deformities, joint instability and dislocations. Like many neuromuscular diseases, the disease can progress to involve respiratory compromise, resulting in pneumonias or respiratory failure. The common cold or flu can be devastating.