...DISEASE CATEGORY: Cancer INDICATION: Neurofibromatosis Cell culture and mouse studies suggest promoting PMP22 expression, dual RUNX1/RUNX3...
...neurofibroma Schwann cells, overexpression of the RUNX1/RUNX3 target PMP22 decreased cell growth compared with normal PMP22...
...RUNX proteins decreased tumor growth compared with vehicle. Next steps could include identifying and testing PMP22...

...INDICATION: Neuropathy Mouse and rat studies suggest inhibiting PMP22 could help treat Charcot-Marie-Tooth 1A (CMT1A) disease...
...disease. In a mouse model of CMT1A, subcutaneous injection of an antisense oligonucleotide (ASO) targeting PMP22...
...Ionis Pharmaceuticals Inc. include identifying and testing undisclosed PMP22 inhibitors in models of CMT1A. TARGET/MARKER/PATHWAY: Peripheral myelin protein 22 (PMP22)...

...growth factor receptor 1 (FGFR1; CD331), cyclin-dependent kinase inhibitor 1A (p21, Cip1; CDKN1A; CIP1) and peripheral myelin protein 22 (PMP22)...

...day for 5 weeks followed by once-daily 5 mg/kg ADX71441 for 4 weeks down regulated PMP22...
...a rare hereditary motor and sensory demyelinating peripheral neuropathy disease caused by overexpression of the PMP22...