Opinion: California’s Rare Disease Council Will Transform Care for People with ALS
California’s recent decision to create a Rare Disease Advisory Council is a milestone that brings new hope to people living ...
Hosted on MSN1d
How logistics shape the success of rare disease trials
Both events are taking place simultaneously 19–20 November in Brussels, Belgium. As interest in rare disease drug development ...
STAT9d
My son’s rare disease was renamed ASMD. Why are people still calling it Niemann-Pick?
Eponyms — diseases named after a person — can cause serious confusion. That is particularly true in one rare disease ...
FiercePharma1d
FDA rejects Intercept's ask for Ocaliva full nod in rare liver disease, but accelerated approval remains
In the wake of an unfavorable advisory committee meeting in September, Intercept Pharmaceuticals’ bid to win full approval ...
ABP Live on MSN6d
This Rare Disease Catches Infants Within First 6 Months, Could Go Unnoticed Until It’s Too Late
By Dr. Kaushik Mandal Rare diseases very often remain in the shadows, as common people have limited awareness about them.
GEN9h
AI Collaboration Between Sanofi and Healx Will Target Novel Rare Diseases Indications
to use its artificial intelligence to identify novel rare disease indications for a discontinued drug asset. Under the terms of the agreement, Sanofi will provide data related to the unnamed drug ...
Warrington Wolves CEO reveals fear after being struck down by rare disease
Karl Fitzpatrick has described his frightening ordeal after being diagnosed with potentially life-threatening Guillain–Barré ...
Cherokee Nation Hosts First Rare Disease Summit to Address Overlooked Health Care Needs
Cherokees deserve help, treatment and hope – especially those who’ve long felt unseen by the health care system due to their ...
YTN4h
Establishment of a basis for supporting the production and seller of medical devices for rare diseases
but the scope of support has expanded as the "Rare Disease Control Act amendment" passed the National Assembly. Ji Young-mi, ...
News on 61d
Tulsa Mechanic, Community Rally To Give 21-Year-Old With Rare Disease Gift Of Adventure
In Tulsa, mechanic Matt Parker rallied the community to support 21-year-old Corbin Norcom, who has a fatal disease, by ...
GlobalData on MSN16h
Intercept loses Ocaliva FDA full approval bid in rare liver disease
Intercept’s US president Vivek Devaraj said the pharma company still believes in the evidence supporting the liver disease ...
The American Journal of Managed Care6d
Rare Disease
Patient input and experiences play a crucial role in advancing rare disease research and therapy development, as they help define the disease, inform clinical trial design, and influence ...