Death after High-Dose rAAV9 Gene Therapy in a Patient with …
Sep 27, 2023 · Duchenne’s muscular dystrophy (DMD) is a fatal, X-linked myopathy caused by mutations in DMD, the large structural gene encoding dystrophin (2.4 Mb of genomic DNA with 79 exons). 1,2 Dystrophin...
Young participant in Pfizer's DMD gene therapy trial dies - Fierce …
May 7, 2024 · A young boy with Duchenne muscular dystrophy (DMD) participating in Pfizer’s phase 2 gene therapy trial has died, the pharma shared in a May 7 letter (PDF) cited by nonprofit Parent Project...
Life Expectancy in Duchenne Muscular Dystrophy - PubMed …
However, our work also provides age-specific estimates of survival and mortality rates over the whole trajectory of a patient with DMD, allowing natural history models to incorporate mortality representative of the global population of patients with DMD.
Boy Dosed with Pfizer DMD Gene Therapy Dies a Year after Phase …
May 8, 2024 · Pfizer has paused enrollment in a Phase III trial assessing its Duchenne muscular dystrophy (DMD) gene therapy candidate fordadistrogene movaparvovec after acknowledging the sudden death of a...
Patient Death Reported in Phase 2 DAYLIGHT Study of Pfizer’s …
May 8, 2024 · A patient in the phase 2 DALIGHT trial (NCT05429372) assessing Pfizer’s investigational gene therapy fordadistrogene movaparvovec (PF-06939926) in boys with Duchenne muscular dystrophy (DMD) has suddenly passed away, the company wrote in a …
Second Death - Pfizer Reports Young Boy's Death After One Year …
May 8, 2024 · A young patient died due to cardiac arrest after receiving Pfizer Inc’s (NYSE: PFE) experimental gene therapy in a mid-stage trial for Duchenne muscular dystrophy (DMD), the company told in a...
DMD gene therapy death exposes risks of treating older patients
May 19, 2023 · Cure Rare Disease has shared a deep dive into the death of the only participant in a gene therapy trial. The nonprofit and its collaborators tied the death of a patient with Duchenne muscular...
Pfizer reports patient death in Duchenne gene therapy study
May 7, 2024 · A young patient died due to cardiac arrest after receiving Pfizer's experimental gene therapy being tested in a mid-stage trial for a muscle-wasting disorder called Duchenne muscular dystrophy...
Pfizer reports fatality in Phase II DMD gene therapy study
May 8, 2024 · Pfizer has reported the death of a patient in the Phase II DAYLIGHT study of experimental gene therapy, fordadistrogene movaparvovec, to treat Duchenne muscular dystrophy (DMD). The trial is designed to assess the safety and …
Case Report of Patient Death Following High-Dose Gene Therapy for DMD
Oct 19, 2023 · In this case study, a patient who had Duchenne muscular dystrophy (DMD) and received high-dose transgene therapy to upregulate cortical dystrophin subsequently developed acute respiratory...