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359,276 Results for "cure rare disease".
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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
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3 min read
Genetown
Chiesi Global Rare Diseases Showcases Ongoing Commitment to Multiple Rare Diseases Communities at ENDO 2024 Meeting Debut
Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people living with rare diseases, announced updates from their rare endocrine disease portfolio with two late-breaking poster presentations and one oral presentation during the Endocrine Society’s ENDO 2024 Meeting that was held June 1-4, 2024, in Boston, Massachusetts.
June 14, 2024
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9 min read
Press Releases
Oxford-Harrington Rare Disease Centre to Host Symposium on Pioneering Research and Innovation in Rare Disease Treatments
September 24, 2024
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4 min read
Press Releases
National Organization for Rare Disorders (NORD) Launches the Living Rare Study - First to Measure Full Scope of Rare Disease Challenges
October 22, 2024
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3 min read
Press Releases
BPGbio Receives FDA Rare Pediatric Disease Designation for its Investigational Treatment for Epidermolysis Bullosa
October 31, 2024
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5 min read
Genetown
Chiesi Global Rare Diseases Announces Publication of Results from Fabry Disease Patient Survey
Chiesi Global Rare Diseases today announced the publication of results from a Fabry disease patient survey in the peer-reviewed Orphanet Journal of Rare Diseases.
April 24, 2024
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4 min read
Press Releases
Monopar Announces Agreement with Alexion, AstraZeneca Rare Disease For Late-Stage Wilson Disease Drug Candidate
October 25, 2024
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8 min read
FDA
FDA Approves First Interchangeable Biosimilar for Two Rare Diseases
Today, the U.S. Food and Drug Administration approved Bkemv (eculizumab-aeeb) as the first interchangeable biosimilar to Soliris (eculizumab) to treat certain rare diseases.
May 28, 2024
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4 min read
Press Releases
MDA Kickstart Program Receives FDA Rare Pediatric Disease Designation and Orphan Drug Designation for Congenital Myasthenic Syndrome
October 24, 2024
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5 min read
FDA
The FDA Needs a Dedicated ‘Center for Rare Diseases’
The agency’s inertia and bureaucratic roadblocks are throttling hope for millions of patients. A new center of excellence would provide a solution.
September 3, 2024
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6 min read
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Henry I. Miller
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