To the Editor:Re “Why Can’t More Children Get the Treatment That Saved My Son’s Life?,” by Elizabeth Currid-Halkett (Opinion guest essay, Feb. 24):Our three children, ages 5 and 7, battle a rare, relentless and ultimately fatal disease called cystinosis.
We recently found hope in the initial phase of a gene therapy clinical trial that was shown to be safe and yielded very promising results — a therapy that could one day save our children’s lives.
Our biggest fear is that it will not be accessible to them or others in desperate need.
We applaud Dr. Marks’s wisdom and perspective.
reviewers’ initial rejection is a cautionary tale of how patient access to lifesaving therapies could be impeded by a narrow interpretation of efficacy.
Persons:
Elizabeth Currid, Currid, Peter Marks, “
Organizations:
Biologics