Males with nonsense mutation in Duchenne muscular dystrophy (DMD) treated with ataluren (Translarna™) experienced a delay in loss of ambulation by more than 5 years when compared to standard of care alone, according to a news release by PTC Therapeutics.
“We are all proud to see Translana’s life-changing effect on males with Duchenne. The [results’] robustness showing both a five-year delay in loss of walking as well as its ability to prolong lung function confirms what we have seen in our clinical trials,” said Stuart W. Peltz, PhD, chief executive officer of PTC Therapeutics. “These results add to the totality of the evidence of Translarna’s benefit for the patients and their families.”
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According to the findings from the 5-year analysis of the Strategic Targeting of Registries and International Database of Excellence (STRIDE) registry, males treated with a combination of standard of care and ataluren had a loss of ambulation at 17.9 years old as a median age. This dropped to 12.5 years old in males treated with standard of care alone.
Moreover, most (80%) males treated with ataluren plus standard of care were still walking at 12 years old. This percentage dropped to 52% in males receiving standard of care alone.
The results also demonstrated a delay in loss of lung function. Males treated with ataluren reached a predicted forced vital capacity lower than 60% at a median age of 17.6 years, compared to 15.8 years for males who did not receive the treatment. This was important since the sub-60% threshold usually indicates the need for starting respiratory physical therapy, the authors said.
According to Filippo Buccella, patient advocate and founder of Parent Project Italy, “We are starting to see more evidence that Translarna can potentially give children many more years of freedom, and this will bring real hope to us parents.”
The results were based on the registries from 241 males in 13 countries who were entered in the STRIDE patient registry over the past 5 years.
STRIDE data show Translarna™ delays loss of ambulation by more than five years in boys with nonsense mutation Duchenne muscular dystrophy. News release. PTC Therapeutics, Inc; September 20, 2021.