أعلنت شركة Sarepta Therapeutics يوم الثلاثاء أن أول حالة وفاة مسجلة مرتبطة بعلاجها الجيني لمرض ضمور العضلات دوشين، Elevidys، حدثت لصبي يبلغ من العمر 16 عامًا.
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Teen Who Received Elevidys Infusion Dies
على مدى العشرين عامًا الماضية، كان من المعروف أن العديد من إجراءات العلاج الجيني تسبب فشل الكبد، وهو ما حدث للطفل. [اقرأ المزيد: الأسئلة الشائعة حول عقار Elevidys المستخدم لعلاج ضمور العضلات دوشين]
The death “represents a severity of acute liver injury not previously reported for Elevidys,” Sarepta noted. The patient had also tested positive for cytomegalovirus (CMV) infection, which can damage the liver and is “a possible contributing factor” to the death, according to the announcement. Sarepta expressed “profound” sadness over the death. [ما هو عدوى الفيروس المضخم للخلايا (CMV)؟]
Sarepta Shares Drop 28%
Sarepta has reported the death to the relevant health authorities and will update Elevidys’ label to “appropriately represent this event,” the biotech noted in its Tuesday release. The company’s stock plunged around 28% as the markets opened on Tuesday to $73.01, down from $101.35 at the previous market close.
يتعلم أكثر: مراجعة التجارب السريرية للعلاج الجيني Elevidys: هل يستحق العلاج بـ Elevidys التكلفة؟
In Which Country and In Which Hospital Did This Sad Incident Occur?
It was reported by Sarepta that the young DMD patient who died was a US citizen and the incident occurred in the US. [Read more: Community Letter]
If sufficient and satisfactory information comes from Sarepta, we will notify all DMD communities that we will make a special news on this subject.