FT Health: The Future of Rare Diseases

There are few treatment options for orphan conditions, which affect only small numbers of patients. But scientists are zeroing in on particular genes to develop therapies that may give some hope

Treatments are being tailored to a specific mutation

Former Genzyme chief helped make ‘orphan’ drugs viable

Nowhere has the value of a single market been seen more clearly

Mayana Zatz’s work on twins shows a genetic component to the birth defect

Recent moves by Beijing could open the door to wider coverage

Genomics England’s progress enhances the quest for new treatments

Fresh ideas for diagnosis, treatment and prevention are being tested, as 2m continue to live with the disease

Conventional treatments developed by Vertex help many sufferers, but they have a hefty price tag