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Maggie’s Pearl, a collaboration between Perlara, Maggie’s Cure, and the Mayo Clinic, announced that the Phase III trial of oral epalrestat therapy in pediatric subjects with PMM2-CDG (formerly, Congenital Disorder of Glycosylation Type 1a) has been green-lighted to transition to open label.

Maggie’s Pearl, a joint venture between Perlara, Maggie’s Cure, and the Mayo Clinic, has started a Phase III clinical treatment trial to evaluate safety, tolerability, and clinical and metabolic improvement of pediatric subjects with PMM2-CDG (formerly known as Congenital Disorder of Glycosylation Type 1a) on oral epalrestat therapy versus a placebo.

Perlara today announced a Mucopolysaccharidosis Type IIIA and Type IIIB PerlQuest partnership with Cure Sanfilippo Foundation, a South Carolina-based 501(c)(3) not-for-profit organization funding research to develop treatments and a cure for Sanfilippo Syndrome.

Perlara, a drug discovery platform company partnering with highly motivated families and drug developers to treat diseases thought too rare to attract the interest of pharmaceutical companies, today announced a collaboration with Harvard Medical School and the Undiagnosed Diseases Network to launch PerlQuests for two rare monogenic neurodevelopmental disorders.

Perlstein’s approach with his start-up, Perlara, is radically different from how most start-ups operate.

Radical Investments is the venture capital firm of serial entrepreneur and impact investor Mark Cuban.

APOL1 kidney disease PerlQuest co-development partnership with Rambam Medical Center in Haifa, Israel, and the laboratory of leading nephrologist and kidney disease researcher Dr. Karl Skorecki.

Glycogen storage diseases (GSD) are monogenic recessive inborn errors in metabolism involving genes that synthesize or degrade glycogen in cells and tissues of the body.

Multiple Sulfatase Deficiency is an ultra-rare monogenic lysosomal disorder caused by mutations in the evolutionarily conserved gene SUMF1