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UnknownReal-world effectiveness of eliglustat in treatment-naive and switch patients enrolled in the International Collaborative Gaucher Group Gaucher Registry.
Not available
S-EPMC7497238 | biostudies-literature
UnknownReal-world outcomes from a series of patients with late onset Pompe disease who switched from alglucosidase alfa to avalglucosidase alfa.
Not available
S-EPMC10834735 | biostudies-literature
UnknownBeyond predicting diagnosis: Is there a role for measuring biotinidase activity in liver glycogen storage diseases?
Not available
S-EPMC9248216 | biostudies-literature
UnknownLower Urinary Tract Symptoms and Incontinence in Children with Pompe Disease.
Not available
S-EPMC5059197 | biostudies-literature
UnknownCase report: Expanding the understanding of the adult polyglucosan body disease continuum: novel presentations, diagnostic pitfalls, and clinical pearls.
Not available
S-EPMC10758020 | biostudies-literature
UnknownSustained immune tolerance induction in enzyme replacement therapy-treated CRIM-negative patients with infantile Pompe disease.
Not available
S-EPMC5621909 | biostudies-literature
UnknownQuantitative whole-body magnetic resonance imaging in children with Pompe disease: Clinical tools to evaluate severity of muscle disease.
Not available
S-EPMC7802624 | biostudies-literature
UnknownAssessment of Cognitive Scales to Examine Memory, Executive Function and Language in Individuals with Down Syndrome: Implications of a 6-month Observational Study.
Not available
S-EPMC4650711 | biostudies-literature
UnknownValidation of the Patient-Reported Outcomes Measurement Information System (PROMIS<sup>®</sup>) physical function questionnaire in late-onset Pompe disease using PROPEL phase 3 data.
Not available
S-EPMC10830974 | biostudies-literature
UnknownImpact of muscular symptoms and/or pain on disease characteristics, disability, and quality of life in adult patients with hypophosphatasia: A cross-sectional analysis from the Global HPP Registry.
Not available
S-EPMC10083387 | biostudies-literature
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