"Most rare diseases lack even one approved treatment. Regulators have tried to encourage drug development, but advocacy groups are having to fight to get the research done."
In 2016 freelance science writer, James Mitchell Crowe had this article published in the British multidisciplinary scientific journal, Nature. The article profiled the work of the Sanfilippo Children's Foundation and the Donnell family while exploring the unique challenges the rare disease community face in getting potential therpies trialled and approved.
