Rollout expected by fall in US for new DMD therapy Duvyzat

ITF Therapeutics — which will be responsible for marketing Duvyzat (givinostat), Italfarmaco’s newly approved Duchenne muscular dystrophy (DMD) therapy, in the U.S — expects the oral medication to be available to eligible adults and children by this fall. For ITF, the U.S.-based rare disease division of Italfarmaco,…

My mind is spinning like the wheels on my son’s power wheelchair in the freshly fallen snow. You did not read that wrong, and it’s not a hypothetical. Here in Nebraska, it snowed this last week of March. It feels like a second winter. As if the spring snow isn’t…

Regulators in France and Italy have given Atamyo Therapeutics the green light to launch a clinical trial testing ATA-200, an investigational gene therapy for children with a type of limb-girdle muscular dystrophy called LGMD2C or LGMDR5. The multicenter, dose-escalation Phase 1b study (NCT05973630) — which will assess…

I received an overwhelming response to my last column, in which I grappled with feelings of uselessness when my facioscapulohumeral muscular dystrophy (FSHD) prevented me from helping in the way I would have liked to do. In this case, I was sitting at home while my wife, Wendy,…

In myotonic dystrophy type 2 (DM2), muscle cells have dysfunctional mitochondria, the cell’s so-called powerhouse that’s key to energy generation. The results imply that treatments to boost mitochondrial function could be beneficial in DM2, the researchers said in “Multi-level profiling unravels mitochondrial dysfunction in myotonic dystrophy…

The U.S. Food and Drug Administration (FDA) has approved Italfarmaco’s givinostat, which will now be marketed as Duvyzat, as an oral suspension to treat people with Duchenne muscular dystrophy (DMD) ages 6 and older. Duvyzat becomes the first nonsteroidal therapy approved in the U.S. for patients with all…

I recently took my three sons with Duchenne muscular dystrophy (DMD) to a hospital in Denver for three days of a clinical trial. One day was for each of them — Max, 18, Rowen, 15, and Charlie, 13 — and then they each had a day of cardiac…

Treatment with givinostat — an oral therapy currently up for approval in the U.S. and Europe — significantly slowed motor function loss among boys with Duchenne muscular dystrophy (DMD) in the Phase 3 clinical trial EPIDYS, according to the full study results. Those results were detailed in a…

Agamree (vamorolone) is now available for Duchenne muscular dystrophy (DMD) patients, ages 2 and older, in the U.S., where it will be dispensed through a specialty pharmacy network. The U.S. Food and Drug Administration (FDA) approved the next-generation steroid, expected to be more tolerable than standard…

I am traveling this week with my three sons who have Duchenne muscular dystrophy (DMD): Max, 18; Rowen, 15; and Charlie, 13. The four of us are in Denver for three days of clinical trial appointments and one day of cardiology appointments. Other Duchenne moms are often surprised…