Tomi Pastinen: Treating All the Not-So-Rare “Rare Disease” Patients at Major Care Centers
Credit: JGI/Jamie Grill / Tetra images Jonathan D. Grinstein, PhD, North American Editor of Inside Precision Medicine, hosts a new...
Rare
Major change for rare disease treatments on way, signals MHRA
New paper published today commits UK to major reform in rare therapies Bold new rulebook for rare therapies is being...
Shunts Safe And Effective For Rare Brain Condition In Elderly, Clinical Trial Concludes | Health
Key TakeawaysA surgically implanted shunt can restore walking ability in elderly people with normal pressure hydrocephalusPatients with shunts could walk...
A Rare Disease Sheds Light on More Common Conditions > News > Yale Medicine
When Pramod Mistry, MD, a Yale Medicine expert in inherited liver diseases, cared for his first patient with Gaucher disease...
Rare disease challenges and potential actions in the Middle East | International Journal for Equity in Health
The findings presented below outline the outcomes of our literature search with respect to challenges, policies, and orphan drug regulations....
Beyond Rare Disease: Wet AMD Gene Therapy Could Reach Millions
As gene therapies continue to transform the rare disease treatment landscape, a new generation is on the cusp of breaking...
