Real-World Clinical Profiles of Patients With Alpha-Mannosidosis: Baseline Evaluations From the SPARKLE Registry
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Submitter Only - Adam Bijou, MD; Presenting Author - Nicole M. Muschol, MD; Co-Author - Nathalie Guffon, MD; Co-Author - Andrea Ballabeni, PhD; Co-Author - Line Borgwardt, MD, PhD; Co-Author - Allan Lund, MD, DMSc; Co-Author - Mercedes Gil-Campos, PhD; Co-Author - Francesca Dona, NA; Co-Author - Julia B. Hennermann, MD;
Meta Tag
Clinical History
Enzyme Replacement Therapy
Lysosomal Diseases
Therapy
Co-Author Nathalie Guffon, MD
Co-Author Andrea Ballabeni, PhD
Co-Author Line Borgwardt, MD, PhD
Co-Author Allan Lund, MD, DMSc
Co-Author Mercedes Gil-Campos, PhD
Co-Author Francesca Dona, NA
Co-Author Julia B. Hennermann, MD
Presenting Author Nicole M. Muschol, MD
Submitter Only Adam Bijou, MD
Keywords
alpha-mannosidosis
clinical profiles
symptoms
neurological system
musculoskeletal system
cognitive disability
facial dysmorphism
heart abnormalities
lung abnormalities
personalized treatment

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