DOSE ESCALATION ENZYME REPLACEMENT THERAPY FOR TREATING ACID SPHINGOMYELINASE DEFICIENCY

The invention relates to a recombinant human acid sphingomyelinase (rhASM) for use in a method of treating an acid sphingomyelinase deficiency (ASMD) in a human subject in need thereof, the method comprising administering to the human subject at least one initial dose of 0.025 mg/kg to 0.05 mg/kg of...

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Bibliographische Detailangaben

Hauptverfasser:	DESNICK Robert J, SCHUCHMAN Edward H, COX Gerald F, MURRAY James M, ANDREWS Laura P
Format:	Patent
Sprache:	eng ; slv
Schlagworte:	HUMAN NECESSITIES HYGIENE MEDICAL OR VETERINARY SCIENCE PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS
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