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One of the top academic centers in the world, conducting a retrospective natural history study for CMT6, the C12orf65 (also referred to as MTRFR) gene.
The Willis Lab has developed a CMT4A rat model developed and owned by HNF to test a gene therapy to treat GDAP1, a recessive CMT4 subtype.
The Saporta CMT Stem Cell Laboratory is a state-of-the-art facility working to develop iPSc’s for CMT4A (GDAP1) and CMT6 (C12orf65, also known as MTRFR gene).
A genomic data information system platform for real-time sharing of CMT genome-level data to advance research.
Principal Investigator Manzoor Bhat, PhD has dedicated his career to the understanding of a rare and fatal type of Inherited Neuropathy called CNTNAP1.
A premiere laboratory animal sourcer, has developed a rat model for two types of CMT - CMT4A-GDAP1 and SORD deficiency
Supporting PK dog studies to advance the HDAC6i program for CMT2A.