Comparing the efficacy of cipaglucosidase alfa plus miglustat with other enzyme replacement therapies for late-onset Pompe disease: a network meta-analysis …
S Shohet, N Hummel, S Fu, I Keyzor… - Journal of …, 2024 - becarispublishing.com
Aim: Late-onset Pompe disease is characterized by progressive loss of muscular and
respiratory function. Until recently, standard of care was enzyme replacement therapy (ERT) …
respiratory function. Until recently, standard of care was enzyme replacement therapy (ERT) …
Validation of the Patient-Reported Outcomes Measurement Information System (PROMIS®) physical function questionnaire in late-onset Pompe disease using …
PS Kishnani, S Shohet, S Raza, N Hummel… - Journal of Patient …, 2024 - Springer
Background The construct validity and interpretation of the Patient-Reported Outcome
Measurement Information System (PROMIS®) Physical Function short form 20a (PF20a) …
Measurement Information System (PROMIS®) Physical Function short form 20a (PF20a) …
[PDF][PDF] Indirect treatment comparison of three enzyme replacement treatments for late-onset Pompe disease: a network meta-analysis with patient-level and aggregate …
S Fu, N Hummel, T Mozaffar, J Castelli… - World Muscle Society …, 2022 - researchgate.net
• Pompe disease is a rare disorder caused by a lack or deficiency of the enzyme acid α-
glucosidase (GAA) that hydrolyses lysosomal glycogen1 and is characterised by …
glucosidase (GAA) that hydrolyses lysosomal glycogen1 and is characterised by …