» German Federal Joint Committee Issues Positive Medical Benefit Rating for Translarna™ in Duchenne Action Duchenne: "PTC Therapeutics, Inc today announced that the Benefit Assessment by Germany’s Federal Joint Committee (G-BA) indicated that Translarna™ (ataluren) provided a benefit for ambulatory patients aged five years and older with nonsense mutation Duchenne Muscular Dystrophy (nmDMD). The G-BA came to the decision that the existing clinical data package presented by PTC provided convincing evidence for the demonstration of a clinically meaningful added benefit. PTC received a 3 in the rating system established under the German pharmaceutical law.
“We are pleased by the positive assessment we received from the G-BA for Translarna. It reflects the medical benefit provided for nonsense mutation DMD patients where there are no other approved therapies,” said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. “We appreciate the G-BA’s recognition of Translarna’s benefit for these patients with such high unmet medical need.”"
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