Overall, Yu and colleagues found that Mila's neurologic measurements stabilized and her seizures were vastly diminished, with no serious adverse events through the first year. Then, Mila received two additional loading doses, followed by maintenance dosing approximately every 3 months. Mila, whose story was first told a year ago by STAT, developed normally until age 3, when her parents noticed that her foot was turning inward, according to the NEJM paper.ĭose escalation started at 3.5 mg, increasing every 2 weeks up to 42 mg. Janet Woodcock, MD, director of the FDA's Center for Drug Evaluation and Research, and Peter Marks, MD, PhD, director of the FDA's Center for Biologics Evaluation and Research, cited that story in an accompanying editorial, saying Yu's work 'illustrate(s) how recently developed technologies permit the delineation of pathways for truly individualized drug development.'

MedPage Today recently reported on a handful of patients and parents who, like Mila, were seeking personalized treatments with ASOs for various conditions. regulatory burden, manufacturing capacity, cost, and reimbursement).' 'his experience indicates that antisense oligonucleotides may deserve consideration as a platform for the rapid delivery of individualized treatments,' Yu and colleagues wrote, cautioning that the approach 'is at present probably scalable to only a limited number of patients, given current limitations on systemic and infrastructural issues (e.g.

Olari by Mila N. Sankale