Agency officials promise fast reviews of new treatments while vowing they will not be a “rubber stamp” for the industry. But ...

Misdiagnosis or underdiagnosis of rare diseases in patients with diagnoses of common diseases can lead to delayed or inappropriate treatments, thereby complicating the management of both rare and ...

Researchers at the Sanford Stem Cell Institute have taken the fight against rare diseases to the lab, to patients’ bedsides ...

In medicine, “rare” is often used to describe conditions that affect relatively few people. But when you work in healthcare ...

Global rare disease policy shifts are reshaping orphan drug development, with major implications for equitable access in the UK, EU and US.

Her son Ben, she learned, had mucopolysaccharidosis (MPS) type 2, or Hunter Syndrome, one of a group of rare diseases that is ...

More than 100 children with rare diseases and their parents participated in a special Patient Support Group Program organised ...

Communication gaps, preventable delays and limited support place significant responsibility on patients on the rare disease journey, a new survey finds. | Ninety percent of patients and caregivers ...

This past spring, a biotech company announced the first use of a new gene-editing technology in people to fix an errant gene that causes a severe immune disorder. In June, a baby born with a ...

"They had happy days on this earth," Azeza Kasham said of her late son, Hiatham, and her son, Gigi, both of whom were ...

The Union Budget has offered relief for rare disease medicines. But families say accessibility and affordability remain major ...

"Beast Games" winner Jeff Allen hopes finding a cure for his son's rare disease will unlock new treatments for other brain conditions.