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. Author manuscript; available in PMC: 2020 May 30.
Published in final edited form as: Prenat Diagn. 2020 Mar 20;40(6):738–745. doi: 10.1002/pd.5678

Table 4.

Therapies for types of LSD in our study

Lysosomal Storage Disease Approved therapies1 Potential therapies in Clinical Trials*
Galactosialidosis Symptomatic and supportive therapy None
SASD Symptomatic and supportive therapy None
Mucopolysaccharidosis VII ERT ERT, Stem Cell Transplant in children and adults
Gaucher ERT and SRT (types I, II and III), none (perinatal lethal form) Stem Cell T ransplant in children and adults
Sialidosis Symptomatic and supportive therapy None
GM1 Gangliosidosis Symptomatic and supportive therapy Stem Cell Transplant in children and adults
Niemann-Pick disease type C SRT Stem Cell Transplant in children and adults
Mucolipidosis II/III Symptomatic and supportive therapy Stem Cell Transplant in children and adults

ERT, enzyme replacement therapy; SRT, substrate reduction therapy.

1

Platt FM, d’Azzo A, Davidson BL, Neufeld EF, Tifft CJ. Lysosomal storage diseases. Nat Rev Dis Primers. 2018 Oct 1;4(1):27.

*

Clinicaltrials.gov accessed on 1/27/2020