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 ...
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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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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 ...
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 ...
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 ...
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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 ...
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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 ...
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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 ...
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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 ...
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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 ...
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 ...