Publications by authors named "Sheela Das"

Objective: To analyze within-group effect sizes of cipaglucosidase alfa plus miglustat (cipa+mig) and alglucosidase alfa (alg) for efficacy, quality of life (QoL), and biomarker variables in ERT-experienced patients.

Background: The randomized, double-blind PROPEL study (ATB200-03; NCT03729362) compared the efficacy and safety of the investigational two-component enzyme replacement therapy (ERT) cipa+mig with alg plus placebo in adults with late-onset Pompe disease (LOPD); 77% of patients had received ERT with alg before study entry (median ERT duration 7.4 years).

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Article Synopsis
  • Cipaglucosidase alfa plus miglustat (cipa + mig) is a new two-part treatment for Pompe disease, assessed in the Phase I/II ATB200-02 study over 48 months.
  • The study involved four adult groups, including both ambulating and non-ambulating patients, who received specific doses of cipa and mig biweekly.
  • Results showed improvements in walking distances and respiratory capacity, especially in ERT-naïve patients, with the treatment generally well tolerated and a safety profile similar to existing therapies.
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Various inclusions have been reported in the past in plasma cells as well as myeloma cells. Most of these represent light chains of immunoglobulins which are concentrated within these cells. Auer rod-like inclusions in myeloma cells have been infrequently reported.

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