Publications by authors named "B Holt"

Cyclosporine A combined with mycophenolate mofetil (CsA/MMF) has become an established regimen for the prevention of graft-versus-host disease (GVHD) following non-myeloablative (NMA) allogeneic hematopoietic stem cell transplantation (alloHSCT). However, the optimal duration of immunosuppression (IS) has not yet been defined and overtreatment is of concern. We hypothesized that time-restricted IS with CsA/MMF would increase the proportion of patients with non-severe GVHD compared to standard-duration IS, thereby resulting in reduction of the relapse rate and improvement of progression-free survival (PFS) and overall survival (OS).

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Article Synopsis
  • Pompe disease results from a deficiency in acid alpha-glucosidase, leading to muscle weakness, respiratory issues, and cardiomyopathy in infants; the only current treatment is enzyme replacement therapy (ERT).
  • A new approach using a Centyrin protein-conjugated short interfering RNA (siRNA) targets the transferrin receptor (CD71) and the GYS1 enzyme to inhibit glycogen synthesis, potentially restoring glycogen balance instead of just degrading it.
  • In tests on a Pompe mouse model, this novel siRNA conjugate effectively reduced GYS1 levels and glycogen accumulation, improving exercise performance, indicating its promise as a therapy for late-onset Pompe disease or in combination with ERT for infants.
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  • Crohn's disease strictures are typically managed with a single endoscopic balloon dilation (EBD), but researchers explored the effectiveness of repeat EBD combined with needle-knife stricturotomy (NKSt) alongside aggressive drug therapy.
  • In a study of 21 patients, those receiving intensive treatment (multiple EBDs and/or NKSt) showed a higher symptom improvement rate (72%) compared to those with a single EBD (40%).
  • The findings suggest that repeat dilations and NKSt are safe and potentially more effective for treating strictures, especially when paired with enhanced drug treatment for inflammation.
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