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 ...
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 ...
pharmaphorum6d
Patient Recruitment for Rare Disease Trials Summit
Recruiting patients for rare disease trials is akin to finding a needle in a haystack, with challenges such as geographical ...
Repurposed FDA-approved drug could uncover potential treatment for rare genetic disorders
In a new study, researchers at McMaster University have identified a potential treatment for Sandhoff and Tay-Sachs ...
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 ...
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 MSN23h
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 ...
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 ...
Treatment to preserve sight in children with rare genetic disorder shows promise in clinical trial
A new treatment that could prevent blindness in children with the CLN2 type Batten disease has been trialed by clinicians and ...
Targeted Oncology1d
Elraglusib Gains FDA Rare Pediatric Disease Designation in Ewing Sarcoma
Elraglusib is undergoing investigation in an open-label, multicenter, phase 1/2 trial for the treatment of patients with ...
STAT10d
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 ...
Business Insider2d
Actuate Announces FDA Rare Pediatric Disease Designation Granted to Elraglusib for Treatment of Ewing Sarcoma
“Receiving rare pediatric disease designation from the FDA underscores the urgent need for new treatment options for patients with EWS and recognizes elraglusib’s transformative potential,” said ...