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Our Mission

To improve the lives of children living with a Lysosomal Storage Disease (LSD).  We remain laser focused on accelerating the discovery and development of treatment options for these diseases with significant unmet medical needs.  

Our current focus is on Acid Sphingomyelinase Deficiency (ASMD), also referred to as Niemann Pick Disease Types A & B (NPA & NPB).

  • Babies born with a Lysosomal Storage Disease each day

    48

  • Identified Lysosomal Storage Diseases (LSD's)

    50

  • 1 in 7700 Children are diagnosed with a LSD

    7700

  • FDA approved treatment options currently exist for ASMD

    0

News & Notes

Inhibition of fatty acid amide hydrolase prevents pathology in neurovisceral acid sphingomyelinase deficiency by rescuing defective endocannabinoid signaling

Clinicopathologic correlations of a case report of a child with NPA

Adeno-associated viral vector serotype 9–based gene therapy for Niemann-Pick disease type A

Our Warriors

  • Jaxon
  • Quinn
  • Jacob
  • Trek
  • Clayton
  • Purnell
  • Corbin