A lovely piece in StatNews recently spotlighted the case of Mila, a child with the rare neurodegenerative genetic disorder Batten disease. Thanks to an experimental drug created by Dr. Timothy Yu and his team at Boston Children’s Hospital, Mila has seen improvement in her symptoms.
The story emphasizes both the possibilities of creating therapies tailored to the specifics of a given patient’s genetic anomalies, but also makes the enormous costs involved in this work very clear.
