On August 25, ITF Therapeutics – the U.S. branch of Italfarmaco – released positive long-term safety and efficacy results for givinostat, a therapy for Duchenne. This treatment, alongside steroids, delayed disease progression and improved key mobility tasks including rising from the floor, climbing stairs, and walking.
Specifically, Givinostat appears to slow down the loss of walking by on average three years. Patients were treated with the drug for on average 1.5 years, suggesting safety over the long term. During this time frame, mostly mild safety issues emerged such as diarrhea. However, the most common severe symptoms were femur fracture and back pain, matching previous clinical results.
For patients with Duchenne, these improvements show promise to build on steroid treatment and slow loss of walking.
Givinostat is not approved in Canada but is approved in the U.S., E.U., and U.K. There are three active Canadian trials for givinostat in Calgary, AB; Vancouver, BC; and Toronto, ON. Defeat Duchenne Canada is following up with ITF Therapeutics for more information and monitoring this process closely.
Read the full press release from ITF Therapeutics below.
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Scientists and medical experts have been tirelessly pursuing diverse approaches to defeat Duchenne muscular dystrophy. From tackling the root cause to alleviating symptoms, the field of research is vast and promising. Explore the various strategies being developed and tested, and gain insights into the future of muscular dystrophy treatment: