Niemann-Pick Type A PerlQuest

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“Perlara’s unique drug discovery platform and business model is a perfect fit for advancing our efforts in discovering existing compounds that can be repurposed, and new novel compounds that can be optimized for treating Niemann-Pick Type A,”

Steven Laffoon
Co-Founder and President of Wylder Nation Foundation

Our Progress

Stage 3 screening of discovery library on nematode model underway.

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PerlQuest updates from our Blog

Developing an NPA patient fibroblast screen

Niemann-Pick Type A (NPA) affects about 1 in 250,000 individuals, with a higher frequency in individuals of Ashkenazi Jewish descent, where it's about 1 in 40,000. NPA is caused by a mutation in the SMPD1 gene, a gene encoding acid sphingomyelinase which, when...

Developmental delay and movement defects in a worm model of Niemann-Pick Type A disease

In December of 2016, Perlara partnered with Wylder Nation to initiate an NPA PerlQuest, a research and drug discovery program for Niemann Pick Type A disease. If you've been following our blog, you've kept up with our fly team's efforts in development of a fly model...

Acid sphingomyelinase (ASM) deficiency in Drosophila causes early larval lethality

A few months ago, Taryn Sumabat blogged about development of a fly model of a Niemann-Pick Type A Disease. Here I’ll remind you of the progress she described, and update you on our more recent work. If you remember, the two main objectives for this phase of our NPA...

Developing a fly model of Niemann-Pick Type A

By Taryn Sumabat This summer at Perlara, we ramped up our efforts to develop a Drosophila model of Niemann-Pick Type A (NPA). NPA is a rare lysosomal storage disease caused by mutations in the gene Smpd1, which encodes an enzyme called acid sphingomyelinase (ASM)....

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