MDF’s work in Care and Cure for myotonic dystrophy has expanded significantly over the past 7 years, and the organization is in a very strong position as we move closer to approved therapies, better diagnosis, access to services and more standardized, high quality care. It is therefore an opportune time to implement a leadership transition that is appropriate to continuing the success of the organization, and Molly White has announced her intent to hand over the reins to a new CEO later this Fall.
Shruti Choudhary’s doctoral work in medicinal chemistry at Duquesne University in Pittsburgh had been focused on cancer and infectious diseases, but when she completed her degree, she turned her attention to myotonic dystrophy. She felt there were plenty of researchers focused on cancer and saw a far greater need to address neurodegenerative conditions.
I met Erich Maurer in early 2013. Before MDF had Facebook groups, it operated a closed online portal that functioned like a bulletin board with threaded discussions. Erich had posted that he was looking for other gay men with DM1, and I eventually sent him a message that I was a gay woman with DM1. And thus a lovely correspondence began.
Kayla Vittek, with her mother, Lisa Harvey-Duren, was a leading advocate for the DM community, regularly appearing in news stories and on television, serving as a Youth Ambassador for Easterseals and participating in the MDA telethon. To remember Kayla, who passed away from heart-related complications in April 2019, Lisa created a fundraising campaign in her memory, and MDF is presenting an outstanding community advocate award at the MDF Annual Conference this Fall.
Many of our community members have joined closed MDF Facebook groups to network, share information and make friends in the DM community. MDF offers private Facebook groups for people with DM1, DM2, JOA, Caregivers, and many more!
A new clinical trial is underway to study the safety, tolerability and pharmacokinetics of ERX-963, a potential therapy for symptoms of excessive daytime sleepiness that may include impaired attention or concentration. The first site for that study, Stanford University, is now recruiting, under the direction of Principal Investigator Jacinda Sampson, MD, PhD.
Dyne Therapeutics has announced a significant investment in the Myotonic Dystrophy Clinical Research Network (DMCRN), which MDF helped launch and expand over the past 5 years, to include new sites across the UK and Europe.
When Luke Bolt was 13, he noticed his hand would lock up when he wrote with a pencil. It was annoying, but it would be several months before he mentioned the problem to his father Steve one night at dinner. When his mother Jodie heard about the problem, myotonic dystrophy was on the top of her list of possibilities.
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