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616,355 Results for "national organization for rare disorders".
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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
·
3 min read
Pharming to be honored as Industry Innovator at National Organization for Rare Disorders (NORD®) 2024 Rare Impact Awards
Pharming Group N.V. is pleased to announce its recognition as an Industry Innovator by the National Organization for Rare Disorders at the Rare Impact Awards on June 8, 2024.
April 24, 2024
·
8 min read
Press Releases
National Organization for Rare Disorders (NORD) Announces $85,000 in Grant Funding for Amyloidosis and Levy-Yeboa Syndrome
October 1, 2024
·
2 min read
Genetown
National Organization for Rare Disorders (NORD) Releases Annual State Report Card, Revealing Trends in Rare Disease Health Care Coverage
The National Organization for Rare Disorders released its annual State Report Card, which grades each U.S. state on critical issues impacting access to care for the 1 in 10 Americans living with a rare disease.
March 6, 2024
·
4 min read
BioCapital
National Organization for Rare Disorders Launches Education Series to Advance Patient Involvement in Rare Disease Drug Development
The National Organization for Rare Disorders launched a new education series in English and Spanish titled, “Rare Disease Drug Development: What Patients and Advocates Need to Know,” designed to help patients and patient advocacy groups understand the drug development process.
October 16, 2023
·
4 min read
Pharm Country
National Organization for Rare Disorders Announces More Than $100,000 in Grant Funding Available for Rare Disease Research
The National Organization for Rare Disorders announced three new grant funding opportunities related to the following rare diseases: Arteriovenous Malformation, Levy-Yeboa Syndrome, and Megacystis Microcolon Intestinal Hypoperistalsis Syndrome.
September 19, 2023
·
2 min read
Complete response letters
FDA Rejects Camurus’ Rare Hormonal Disorder Treatment Due to Manufacturing Problems
The regulator cited deficiencies at a third-party manufacturing facility. Camurus is seeking approval for its extended-release subcutaneous formulation of octreotide, which would allow more convenient once-monthly dosing for patients with acromegaly.
October 22, 2024
·
2 min read
·
Tristan Manalac
Pharm Country
Coalition to Cure Calpain 3 in Partnership with the National Organization for Rare Disorders (NORD®) Launches LGMD2A/Calpainopathy Registry
Coalition to Cure Calpain 3 in partnership with the National Organization for Rare Disorders launched a study with global reach to research Calpainopathy, a rare form of muscular dystrophy that causes progressive muscle weakness and wasting.
September 21, 2023
·
3 min read
Lone Star Bio
Using AI to Improve Diagnosis of Rare Genetic Disorders
Diagnosing rare Mendelian disorders is a labor-intensive task, even for experienced geneticists. Investigators at Baylor College of Medicine are trying to make the process more efficient using artificial intelligence.
April 25, 2024
·
5 min read
Business
Trethera Announces Presentation at the Rare Neuroimmune Disorders Symposium Annual Meeting
October 15, 2024
·
4 min read
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