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628,858 Results for "national organization for rare disorders".
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Rare diseases
Rallybio Pivots as Antibody for Rare Bleeding Disorder Flunks Dosing Trial
The company is dropping its former lead molecule in favor of another antibody, RLYB116, which is being developed for a variety of rare autoimmune disorders.
April 8, 2025
·
1 min read
·
Dan Samorodnitsky
Press Releases
National Organization for Rare Disorders (NORD®) Issues New Report on Life-Saving Newborn Screening Programs
February 11, 2025
·
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
Tracey Sikora Joins the National Organization for Rare Disorders (NORD®) as Vice President of Research & Clinical Programs
December 12, 2024
·
3 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
·
3 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
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
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
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
Press Releases
Ontario Brain Institute partners with EpiSign to advance diagnosis of rare genetic disorders
March 26, 2025
·
3 min read
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