Latest Advances

  • Condition: Rapidly Progressive Lysosomal Acid Lipase Deficiency in Infants
  • Journal: Orphanet journal of rare diseases
  • Treatment Used: Sebelipase Alfa Enzyme Replacement Therapy
  • Number of Patients: 19
  • Published —
The study researched the outcomes of sebelipase alfa enzyme replacement therapy for treating rapidly progressive lysosomal acid lipase deficiency in infants.
  • Condition: Lysosomal acid lipase deficiency
  • Journal: Orphanet journal of rare diseases
  • Treatment Used: Enzyme replacement therapy
  • Number of Patients: 2
  • Published —
The study researched the effects of enzyme replacement therapy for lysosomal acid lipase deficiency.
Showing 1-2 of 2