Retrotope Presents Data Demonstrating Arrest of Disease Progression with Treatment of RT001 in Patients with Infantile Neuroaxonal Dystrophy (INAD)
Retrotope today reported new data that demonstrate arrest of disease progression with treatment of RT001 in patients with infantile neuroaxonal dystrophy (INAD). The data were presented at the National Organization for Rare Disorders (NORD), Rare Diseases & Orphan Products Breakthrough Summit® on October 15-16, 2018, in Washington, D.C.
“INAD is a rare and devastating disease that strikes the very young and there are no current available treatments,” said Peter G. Milner, M.D., Chief Medical Officer of Retrotope. “The two patients enrolled in an expanded access program and presented here have been treated with RT001 for more than one year and six months, respectively. Both show disease arrest and some improvement in several components contained in the INAD rating scale. In the meantime, we are preparing to initiate a pivotal study of RT001 in INAD patients with the objective of confirming its efficacy and safety in this patient population.”
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