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 ...
Clinical trial shows positive results for potential treatment to combat a rare immune-mediated disease
An international, Phase III clinical trial led by investigators at Mass General Brigham could improve the treatment of a rare ...
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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 ...
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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 ...
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 ...
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 ...
GEN18h
AI Collaboration Between Sanofi and Healx Will Target Novel Rare Diseases Indications
Healx will use its AI-based drug discovery tech to analyze proprietary Sanofi compound data and identify potential rare disease targets.
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 ...
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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 ...
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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 ...
Rare disease support: How to get help, resources, manage symptoms, find doctors
Are you battling a rare disease or think you might be suffering from one? If so, there are resources to get help from ...
Developing tailor-made treatments for ultra-rare disease patients: Research provides a roadmap
In a new study, a team of researchers, including Anneliene Jonker, has published, in Nature Reviews Drug Discovery, the first ...