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clinical trial

Phase 3 AFFINE Study of Novel Gene Therapy for Hemophilia Reopens Recruitment

The Pfizer and Sangamo Therapeutics phase 3 AFFINE study assessing the efficacy and safety of giroctocogene fitelparvovec, a novel gene therapy for patients with moderately severe to severe hemophilia A, has reopened recruitment. The reopening, announced by news release, will resume enrollment this month and dosing is expected to restart in October. Giroctocogene fitelparvovec is…

NMOSD Prognosis

Autologous Hematopoietic Stem Cell Transplantation Shows Promise for Severe NMOSD

For patients with severe neuromyelitis optica spectrum disorder (NMOSD), autologous hematopoietic stem cell transplantation (AHSCT) may be used as an alternative to traditional immunotherapies, according to a systematic review and meta-analysis. These data indicated that the Extended Disability Status Scale score prior to and following AHSCT had a pooled standardized mean difference of -0.81 (95%…

DMD history

Siblings of Patients with DMD More Concerned About Work Than Their Peers

The siblings of patients with Duchenne muscular dystrophy (DMD) were more likely to concentrate on DMD-linked desires and aspirations associated with work, intimacy, self-image, community, family, and living with discomfort than a cohort of peers of similar ages, a recent study suggests. The results also showed that the siblings of patients with DMD were more focused…

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