Rhizomelic Chondrodysplasia Punctata (RCDP)
RCDP is an ultra-rare pediatric genetic disorder with an estimated prevalence of 1 in 100,000. The disease results exclusively from a plasmalogen lipid deficiency caused by mutations in genes involved in plasmalogen biosynthesis.
MLD is developing plasmalogen precursor PPI-1011 for the treatment of RCDP. PPI-1011 is the world’s first commercially viable, synthetic ether plasmalogen drug. More information on PPI-1011, RCDP, and the complete clinical development program can be found at www.rhizotrial.org.