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370,187 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
Funding
AIRNA Hits $155M Series B Haul To Advance RNA Editor for Rare Genetic Disease
AIRNA’s lead candidate AIR-001 works by correcting the most common pathologic mutation driving the rare disease alpha-1 antitrypsin deficiency.
April 1, 2025
·
2 min read
·
Tristan Manalac
Press Releases
CTNNB1 Foundation awarded €1M as Slovenia backs nonprofit gene therapy - a first step toward equitable cures for rare diseases
March 31, 2025
·
1 min read
FDA
Makary Discusses Expedited Rare Disease Approvals Pathway, ‘Public Distrust’ in New Interview
In an interview with former
Fox News
journalist Megyn Kelly, FDA Commissioner Marty Makary introduced a new mechanism-driven pathway that could be leveraged by rare disease therapies while saying that autism could potentially be driven by certain environmental factors.
April 21, 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
Press Releases
Cure Rare Disease Appoints Brittany Stineman as Chief Advancement Officer
December 18, 2024
·
2 min read
Rare diseases
Glycomine Gets $115M Series C for Rare Disease, Aims for Phase II Launch Mid-Year
The raise will go toward trialing the company’s lead drug for phosphomannomutase-2 congenital disorder of glycosylation, a rare disease that affects the entire body and produces a wide range of symptoms.
April 16, 2025
·
2 min read
·
Dan Samorodnitsky
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
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
Child’s Cure Genetic Research collaborates with Matica Bio to Drive Innovation in Pediatric Rare Disease Therapies
January 7, 2025
·
3 min read
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