Celebrating milestones of DMD siblings brings mixed emotions

In a large family, there is always something to celebrate. I have seven children whose ages range from 15 months to 21 years old. Last week, we attended opening night at the theater, baseball games, a first communion, and a belated birthday celebration. Next week, we have another opening night…

IPS Heart has received rare pediatric drug designation for its GIVI-MPC and ISX9-CPC, two experimental single-course stem cell therapies for Duchenne muscular dystrophy (DMD). The designation is given by the U.S. Food and Drug Administration (FDA) to spur treatments for any rare disease that can be serious or…

Having reached an agreement with the U.S. Food and Drug Administration (FDA), NS Pharma will be launching a Phase 2 clinical trial to evaluate its investigational exon 44 skipping therapy for Duchenne muscular dystrophy (DMD). Trial details will be forthcoming once enrollment is imminent, according to NS Pharma,…

As I’ve tried to decide what part of Duchenne muscular dystrophy (DMD) to write about this week, I can’t seem to come up with the amount of focus necessary to do so. Instead, Duchenne is always there, all the time. Does that happen to anyone else? Duchenne can take…

The U.S. Food and Drug Administration (FDA) has granted fast track designation to RGX-202, a one-time gene therapy for Duchenne muscular dystrophy (DMD) that is in early clinical trials. The FDA gives this designation to investigational treatments that have the potential to address unmet clinical care needs for…

Each of us dealing with facioscapulohumeral muscular dystrophy (FSHD) is on a slightly different trajectory. For some, the disease is a series of small losses that level out to a period of relative stability. For others, the disease is a steady, steep decline. For some, the disease remains confined…

Levels of titin, a muscle protein, were found to be elevated in the urine of boys with Duchenne muscular dystrophy (DMD) — making it a potential, novel, non-invasive biomarker for the genetic disease, a study demonstrated. Using a standard DMD mouse model, a lack of dystrophin, the protein missing…

While very serious and sometimes grim, the prognosis today for Duchenne muscular dystrophy (DMD) is the best it has ever been. This is because so many new therapies are in the clinical trial process, and some have already been approved. As a result, parents of children…

Microdystrophin gene therapy effectively maintained long-term heart function in a mouse model of severe Duchenne muscular dystrophy (DMD), a study has found. The treatment prevented scar formation and inflammation in heart tissue, and maintained normal heart function over 18 months. These findings support the ongoing clinical trials evaluating…

Note: This column contains spoilers for the series finale of the Netflix TV show “Dead to Me.” As I write this, the date is April 3. I’ve just survived the longest and most helpless week of my life. Two Sundays ago, my support worker, Glenda, suddenly fell ill from…