-
To me, a wheelchair is a symbol of improved function, not disability
Everyone recognizes the blue and white signs featuring a figure in a wheelchair that adorn certain parking spots. I call him the wheelchair guy, and he allows me to park close to public building entrances. The sign is an international symbol of disability. Of course, many disabled people are able to walk, but a wheelchair…
-
Beyond Approval: Why Drug Development Doesn’t End at FDA Sign-Off
For many neuromuscular community members — and clinicians — who are waiting for a new treatment, a drug’s approval by the US Food and Drug Administration (FDA) marks the finish line. Years of discovery, testing, and clinical trials can result in a regulatory green light and, finally, patient access. But for researchers, FDA approval may…
-
When school is out, my sons with DMD get into their summer groove
This past winter in rural Nebraska was mild, with no real snow and only a handful of very cold weeks. But as it has gotten warmer and the grass starts turning green, I very much appreciate the arrival of spring. My children, who will be out of school for summer break by mid-May, have begun…
-
How MDA Summer Camp Influenced the Direction of My Life
Bio: Rebecca Hume is a Senior Specialist Writer for Quest Media. She graduated from Slippery Rock University and worked in the Pennsylvania Medicaid Waiver Program for more than 10 years before coming to MDA. Rebecca was an MDA Care & Clinical Services Specialist and MDA Camp Director prior to accepting her role as a Quest…
-
Upsher-Smith launches new DMD support websites for Kymbee treatment
Upsher-Smith Laboratories has launched two new websites to support people with Duchenne muscular dystrophy (DMD) who are taking its recently introduced corticosteroid therapy Kymbee (deflazacort). One website is designed specifically for caregivers of children with DMD. It offers tips for talking with healthcare providers about treatment, along with a step-by-step guide on how to start…
-
Clinical Research Alert: Clinical Study for Adults with Late-Onset Pompe Disease (LOPD)
Researchers at Shionogi Inc. are conducting a phase 2 clinical trial for adults with late-onset Pompe disease (LOPD) who are receiving standard-of-care enzyme replacement therapy (ERT). The study will evaluate the safety, effectiveness, and processing in the body of the investigational drug S-606001 when used as an add-on to ERT. S-606001 blocks an enzyme involved…
-
Exploring other mobility devices when a walker is no longer sufficient
Last week was a busy one for me. In addition to my normal activities, I took field trips on Tuesday and Thursday, looking for solutions to some of my recent struggles related to facioscapulohumeral muscular dystrophy (FSHD) and other health issues. I’m really struggling to get around our home using my walker. I get partway…
-
Collaboration aims to improve design of FSHD clinical trials
A new collaboration aims to improve the design of clinical trials testing potential treatments for facioscapulohumeral muscular dystrophy (FSHD). The FSHD Society, a patient-driven advocacy organization, is teaming with two other groups — Solve FSHD, a venture philanthropic organization focused on speeding the development of new FSHD treatments, and the FSHD Clinical Trial Research Network, …
-
Pompe Disease and the Real Story Behind Dr. Stonehill, John Crowley, and ‘Extraordinary Measures’
“Don’t hope for a miracle. Make one,” is the powerful tag line from “Extraordinary Measures,” a 2010 dramatic film starring Brendan Fraser, Harrison Ford, and Keri Russell. John Crowley delivered the keynote address at the 2026 MDA Clinical & Scientific Conference. The movie is inspired by the true story of John and Aileen Crowley, a…
-
MDA Ambassador Guest Blog: My Pompe Journey
Kathryn Arvidson champions accessible mental health treatment for people with disabilities. Kathryn was diagnosed as a child with a rare degenerative neuromuscular disorder, Pompe disease, that impacts her mobility and pulmonary functioning. Kathryn earned a master’s degree in social work and served as a Licensed Clinical Social Worker and supervisor for licensed professional counselors in…
