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DMD treatment SMT-M01 gets FDA orphan drug, rare disease tags
SMT-M01, a Duchenne muscular dystrophy (DMD) treatment, was granted orphan drug and rare pediatric disease designations by the U.S. Food and Drug Administration (FDA). The cell replacement therapy from Somite Therapeutics uses artificial intelligence (AI) and large, complex data sets to improve production of multiple human cell types. The company plans to begin clinical trials…
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PPMD Contributes to National Academies Report on Rare Disease Regulatory Processes
We are proud to highlight the National Academies of Sciences, Engineering, and Medicine (NASEM) Consensus Study Report: Regulatory Processes for Rare Disease Drugs in the United States and European Union – Flexibilities and Collaborative Opportunities, with PPMD’s President and CEO Pat Furlong serving on the committee that helped shape its findings. This report outlines crucial…
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Setting Up A Successful School Year
Setting Up A Successful School Year PPMD is here to make sure that families within the Duchenne and Becker community have the necessary tools to advocate for their child not only in medical settings, but at school, as well. Together with you and your school’s staff, we want to ensure your child has a successful…
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PPMD Community Webinar Recap: Overview of DUVYZAT and Access Resources
On September 18, 2024, PPMD hosted an informative community webinar featuring representatives from ITF Therapeutics, PPMD, and the Little Hercules Foundation. The session focused on DUVYZAT (givinostat), a recently approved HDAC inhibitor for the treatment of Duchenne, and the support programs available for patients and families. ITF Therapeutics provided an overview of DUVYZAT, explaining its…
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Overcoming mental hurdles as I transition to using a walker
I’ve had a walker with wheels for a decade. I got it after back surgery in 2014 and used it for a month or so. As a blind person with facioscapulohumeral muscular dystrophy (FSHD), I found it much more difficult to maintain my orientation without direct touch. I was also running the walker into walls,…
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PPMD & Global Patient Advocacy Groups Award BIND Project $113,000 to Further Brain Involvement Research
PPMD is thrilled to announce that we have collaborated with a number of patient organizations around the globe to provide Leiden University Medical Center (LUMC), together with the Duchenne Data Foundation (DDF), funding to further continue deep phenotyping of the brain in Duchenne and Becker muscular dystrophy. This research project, set to commence on October…
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Why patient advocacy is important for those of us with Duchenne
Last Saturday, I had the honor of participating in the third Singapore Health Patient Advocate Connection event (SPACe) hosted by the SingHealth Patient Advocacy Network (SPAN). As a cast member of the incredible SPANtastic Theatre plenary skit team, I not only had the chance to perform, but also to experience something deeply transformative. Although I’ve…
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Losmapimod fails to significantly improve FSHD outcomes: Study
Losmapimod, an investigational medication from Fulcrum Therapeutics, failed to significantly outperform a placebo at improving upper limb function and other clinical measures in people with facioscapulohumeral muscular dystrophy (FSHD), according to top-line data from the REACH Phase 3 trial. Participants given losmapimod did see improvements over 48 weeks, but the placebo group didn’t show a…
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Navigating Access & Resources
Navigating Access & Resources Navigating insurance access for medical care such as approved therapies or equipment can be daunting. Parents, patients, and healthcare providers spend hours putting together documents and speaking on the phone trying to convince payers to approve what is needed to keep us, our children, or our patients, as healthy as possible.…
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A friend in the DMD world makes a difference in my self-care
In my recent columns, I’ve shared several challenges we’ve faced in the past months of my family’s journey with Duchenne muscular dystrophy (DMD). My accessible van will be in the shop for the foreseeable future. I’ve returned to work after over a decade at home as a mom to several children and a caregiver to…
