A rare disease carried by ticks has been detected in Lake County thanks to the help of the Illinois Department of Public ...

Fabry Disease is a rare X-linked genetic lysosomal storage disorder whereby there are excessive fat [lipid] deposits on the tissues of the patient. The many parts of a person's body affected by this ...

Scientists finally know how a crucial fat molecule in the brain manages to survive while helping break down other fats. Are ...

During my “season of PH,” I’ve met new friends who want to learn about the disease and how they can support me. Those are the ones worth keeping around. They might not completely understand what I’m ...

The regulator agreed to allow Sangamo Therapeutics to use data to seek accelerated approval for its Fabry gene therapy ...

Also, Colorado State U. received $50 million to help build a new engineering facility, and the Stuart Foundation pledged $30 ...

Fifty years ago, a memorial fund for research in Reye’s disease was being established at Albany Medical College.

We think Biogen's neurology, immunology, and focus on rare diseases support a narrow moat. Biogen's strategy has its roots in the 2003 merger of Biogen (Avonex for treating multiple sclerosis, or MS) ...

AvenCell Therapeutics announced that it raised $115 million in a Series B round. The company is developing CAR-T therapies ...

You'd hardly know it with the recent streak of summer weather, but we're weeks away from colder days when respiratory viruses ...

Over 10,000 rare diseases are currently affecting more than 30 million Americans. This article, written in partnership with members of the Rare Disease Compliance Consortium, focuses on the role of ...

BNAC’s strongly patient-driven Advisory Council convinced Zivadinov and colleagues to consider studying this rare form of MS. The council’s chair, Larry Montani, had watched two of his siblings ...