11 results on '"Lindschau, Mona"'
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2. Clinical benefit on the skeletal system in a MPS IH patient after hematopoietic stem cell transplantation and long-term enzyme replacement therapy
3. A phase I/II clinical trial of JR-441 for treatment of Sanfilippo syndrome type A (MPS IIIA)
4. Exostosin-like 3 (EXTL3)-related neuro-immuno-skeletal dysplasia syndrome: Comparison of two siblings treated and not treated with hematopoietic stem cell transplantation
5. Experiences and challenges of pregnancies in a patient with mucolipidosis type III
6. Hip dysplasia in Hurler syndrome: A retrospective analysis of longitudinal data from neonatal hip screening to long-term follow-up
7. Klinischer Verlauf bei Patienten mit Mukopolysaccharidose und Spinalkanalstenose mit Rückenmarkskompression
8. Updated results of Transpher B: multicenter, single-dose, phase 1/2 clinical trial of ABO-101 for Sanfilippo syndrome type B (mucopolysaccharidosis IIIB)
9. Hematopoietic stem cell transplantation in a patient with mucolipidosis type II - A five-year follow-up
10. Successful ambroxol treatment in Gaucher disease type 2: Age-appropriate neurocognitive development after one year of follow-up
11. eP214 - Updated results of Transpher B: multicenter, single-dose, phase 1/2 clinical trial of ABO-101 for Sanfilippo syndrome type B (mucopolysaccharidosis IIIB)
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