A young boy died in a trial for Pfizer’s experimental gene therapy for Duchenne muscular dystrophy, the company told patient advocates Tuesday.
The boy was enrolled in Daylight, a trial studying the treatment in boys aged 2 or 3. The boy had received the therapy early last year, Pfizer told the advocates in a note posted online by Parent Project Muscular Dystrophy.
Pfizer said the boy died of cardiac arrest, but that it was still working to understand what happened and the exact cause.
CHICAGO — Progression-free survival (PFS) was significantly higher with belantamab mafodotin (belamaf; Blenrep) combined with pomalidomide (Pomalyst) and dexamethasone (BPd), compared with standard-of-care pomalidomide, bortezomib
Psilocybin is under clinical development by Filament Health and currently in Phase I for Treatment Resistant Depression.
The FDA said Tuesday that it is unconvinced the benefits of Geron’s imetelstat outweigh the risks in certain anemic myelodysplastic syndrome (MDS) patients who are
