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366,422 Results for "cure rare disease".
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Press Releases
NephCure Celebrates 25 Years Closer to a Cure for Rare Kidney Disease
February 6, 2025
·
3 min read
Press Releases
Cure Rare Disease Appoints Brittany Stineman as Chief Advancement Officer
December 18, 2024
·
2 min read
Rare diseases
How SpringWorks Took Pfizer’s Rare Disease Ghost Drug and Got It to Patients
SpringWorks Therapeutics sprung out of Pfizer’s storeroom, when a rare disease advocacy group pushed to keep a program for neurofibromatosis alive. This method could work for “every rare disease under the sun,” advocates say.
February 26, 2025
·
5 min read
·
Annalee Armstrong
Press Releases
Cure Rare Disease Awarded $5.69 Million CIRM Grant to Advance Gene Therapy for Spinocerebellar Ataxia Type 3
February 7, 2025
·
3 min read
Press Releases
Quoin Pharmaceuticals Launches ‘NETHERTON NOW’ Campaign to Raise Awareness of Netherton Syndrome, a Rare Genetic Disease with No Approved Treatment or Cure
February 4, 2025
·
7 min read
Press Releases
Child’s Cure Genetic Research collaborates with Matica Bio to Drive Innovation in Pediatric Rare Disease Therapies
January 7, 2025
·
3 min read
Approvals
Mirum Establishes Liver Expertise With First FDA Nod for ‘Very Rare’ Disease
Ctexli’s approval further entrenches Mirum as a leader in rare liver diseases, alongside its cornerstone product Livmarli and upcoming drug volixibat.
February 24, 2025
·
2 min read
·
Tristan Manalac
Business
Cure Rare Disease Expands Leadership Team and Board of Directors to Advance Mission
Cure Rare Disease, a clinical-stage nonprofit biotechnology organization, announces the addition of a Chief Advancement Officer to its leadership team and welcomes three new members to its board of directors.
June 17, 2024
·
3 min read
Rare diseases
How To Mine Biopharma’s IP Storeroom for Rare Disease Drugs, Just Like SpringWorks
SpringWorks Therapeutics is the perfect case study for rescuing a discontinued assets. It’s time to repeat the process for every rare disease, experts say.
February 26, 2025
·
5 min read
·
Annalee Armstrong
Press Releases
BioMark Diagnostics Underscores Commitment to Rare Disease Research on Rare Disease Day 2025
BioMark is pioneering liquid biopsy solutions for challenging conditions and championing equity in diagnosis
February 28, 2025
·
3 min read
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