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 ...
GEN7h
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 ...
Business Insider1d
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 ...
pharmaphorum5d
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 ...
Actuate announces FDA rare pediatric disease designation to elraglusib
Actuate Therapeutics (ACTU) announced that the U.S. Food and Drug Administration, FDA, has granted rare pediatric disease designation to ...
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 ...
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 ...
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 ...
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 ...
Hosted on MSN23h
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 ...
‘Curse stops with us’: Sad update after Brisbane dad’s rare diagnosis
A Brisbane father has died after being diagnosed with an extremely rare and incurable disease that he dubbed the family ...