Associated tags: Patient, NORD, Disease, Pharmaceutical industry, National Organization for Rare Disorders, Rare, Research, FDA
Locations: PENNSYLVANIA, NORD, MASSACHUSETTS, CHICAGO, ILLINOIS, RARE, MARYLAND, DELAWARE, INDIANA, NEVADA, GEORGIA, IDAHO, NORTH CAROLINA, MISSISSIPPI
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Pharmaceutical industry QUINCY, Mass., May 1, 2024 /PRNewswire/ -- The National Organization for Rare Disorders (NORD) announced today that Pamela K. Gavin has been named CEO, effective immediately. She succeeds Peter L. Saltonstall, who announces his retirement and served as President and CEO since 2008. After a transition period, Saltonstall will continue in an advisory role with NORD.
Key Points:
- QUINCY, Mass., May 1, 2024 /PRNewswire/ -- The National Organization for Rare Disorders (NORD) announced today that Pamela K. Gavin has been named CEO, effective immediately.
- Driven by a family connection to rare disease, Gavin joined the company in 2012 as NORD's first Chief Operating Officer.
- "I want to thank the members and Board of NORD for enabling me to continue my work for the rare disease community in this new role," stated Gavin.
- High resolution photos of Pamela Gavin for media use:
Pamela Gavin Photo 1 (Pamela Gavin named CEO of the National Organization for Rare Disorders; courtesy: National Organization for Rare Disorders)
Pamela Gavin Photo 2 (NORD's new CEO, Pamela Gavin, with her nephew, Trevor, who was diagnosed with a rare disease at age 2; courtesy: Pamela Gavin)
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Pharmaceutical industry "Through science and advocacy, they are transforming the lives of those impacted by rare diseases, paving the way for new possibilities in treatment and care."
Key Points:
- "Through science and advocacy, they are transforming the lives of those impacted by rare diseases, paving the way for new possibilities in treatment and care."
- "Having witnessed my sister's journey, I know the challenges rare disease families face are not just physical but also emotional, mental, and financial.
- In addition to the individual award recipients, the Rare Impact Awards are also honoring companies that have developed treatments changing the lives of those with rare diseases.
- Regeneron Pharmaceuticals for Veopoz™: The first and only treatment indicated specifically for CHAPLE disease, a rare hereditary immune disease.
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Wednesday, April 17, 2024
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Pharmaceutical industry JACKSON, Miss., April 16, 2024 /PRNewswire/ -- Today, patients and families in Mississippi's rare disease community celebrated Governor Tate Reeves' signing of SB 2156, which officially establishes a Rare Disease Advisory Council (RDAC) in the state of Mississippi.
Key Points:
- The RDAC will comprise of a diverse group of rare disease stakeholders including patients, caregivers, healthcare providers, researchers, and members of pharmaceutical and health insurance companies.
- "Our state remains committed to supporting those living with a rare disease, and The Mississippi Rare Disease Advisory Council will help to accomplish exactly that.
- "It has been my pleasure to lead the Mississippi Rare Disease Advisory Council bill for the last several years in the House.
- Aaron Blocker, a patient living with a rare disease in Mississippi said, "The establishment of the Rare Disease Advisory Council in Mississippi stands as a monumental victory for patients like myself across our state.
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Health insurance QUINCY, Mass., March 6, 2024 /PRNewswire/ -- The National Organization for Rare Disorders (NORD) 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. This year's State Report Card highlights state policies that can jeopardize health care coverage, access, and affordability for rare disease patients, including limitations on Medicaid enrollment, inadequate state-regulated insurance, and failure to protect against high out-of-pocket prescription drug costs.
Key Points:
- NORD's State Report Card reveals grades on state policies that could jeopardize health care coverage, access and affordability for rare disease patients
QUINCY, Mass., March 6, 2024 /PRNewswire/ -- The National Organization for Rare Disorders (NORD) 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.
- This year's State Report Card highlights state policies that can jeopardize health care coverage, access, and affordability for rare disease patients, including limitations on Medicaid enrollment, inadequate state-regulated insurance, and failure to protect against high out-of-pocket prescription drug costs.
- These types of plans offer inadequate insurance coverage, typically failing to provide essential health benefits required by the Affordable Care Act and are allowed to deny coverage based on pre-existing conditions.
- One means of advancing favorable rare disease policies at the state level is through each state establishing their own Rare Disease Advisory Council (RDAC) .
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Pharmaceutical industry WASHINGTON, Oct. 16, 2023 /PRNewswire/ -- Today, the National Organization for Rare Disorders (NORD®) 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.
Key Points:
- WASHINGTON, Oct. 16, 2023 /PRNewswire/ -- Today, the National Organization for Rare Disorders (NORD®) 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.
- New education series in English and Spanish: "Rare Disease Drug Development: What Patients and Advocates Need to Know.
- "The goal of this educational series is to engage the rare disease community on their essential role in the drug development process and ultimately help produce more and better treatments for rare disease," said Rebecca Aune, Director of Education Programs at the National Organization for Rare Disorders.
- The first modules, on "Drug Development Process", "Stakeholder Roles in Drug Development" and "Natural History Studies," are now available for free at learn.rarediseases.org .
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Thursday, October 12, 2023
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Management WASHINGTON, Oct. 12, 2023 /PRNewswire/ --
Key Points:
- Join the National Organization for Rare Disorders (NORD) Oct. 15-17, 2023 in Washington for the
Rare Diseases and Orphan Products Breakthrough Summit.
- This event brings together rare
tackle the most pressing issues facing America's rare disease community.
- more than 7,000 rare diseases that impact 1 in 10 Americans.
- From small towns to large cities, more than 30 million
Americans have a rare disease.
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Thursday, September 21, 2023
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Nursing Additionally, the study will help identify individuals with Calpainopathy who might be willing to take part in other research studies or clinical trials.
Key Points:
- Additionally, the study will help identify individuals with Calpainopathy who might be willing to take part in other research studies or clinical trials.
- While the original registry served primarily to contact Calpainopathy patients, the new registry will have the capability to collect longitudinal data by having participants periodically update their information.
- "The LGMD2A/Calpainopathy Registry will provide a complete picture of each patient's experience with this disease," shares Dr. Jennifer Levy, C3 Scientific Director.
- "This new study has tremendous promise as a strong partnership that engages the patient community and addresses current knowledge gaps for Calpainopathy.
Retrieved on:
Tuesday, September 19, 2023
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Pharmaceutical industry DANBURY, Conn., Sept. 19, 2023 /PRNewswire/ -- The National Organization for Rare Disorders (NORD®) announced three new grant funding opportunities related to the following rare diseases: Arteriovenous Malformation (AVM), Levy-Yeboa Syndrome (LYS), and Megacystis Microcolon Intestinal Hypoperistalsis Syndrome (MMIHS).
Key Points:
- The National Organization for Rare Disorders (NORD) announces three new research opportunities for AVM, LYS, and MMIHS.
- NORD's Research Grants Program provides seed-money grants to qualified investigators for scientific and clinical research into diseases for which there are few other sources of funding.
- With funding by the Maxwell Family, NORD is accepting applications for one grant of up to $40,000 US, for scientific and/or clinical research studies related to Levy-Yeboa syndrome (LYS).
- To learn more or submit to the 2023 NORD Research Grants Cycle, visit rarediseases.org/advancing-research/request-for-proposals/
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Nursing WASHINGTON and ALBANY, N.Y., July 6, 2023 /PRNewswire/ -- The Galactosemia Foundation and the National Organization for Rare Disorders (NORD)® today announced they have jointly published the galactosemia community's first "Voice of the Patient" report.
Key Points:
- WASHINGTON and ALBANY, N.Y., July 6, 2023 /PRNewswire/ -- The Galactosemia Foundation and the National Organization for Rare Disorders (NORD)® today announced they have jointly published the galactosemia community's first "Voice of the Patient" report.
- This report provides a comprehensive overview of patient and caregiver experiences with this rare metabolic disease, which currently has no approved treatment options.
- The report follows a historic Externally Led Patient-Focused Drug Development (EL-PFDD) meeting hosted by the Galactosemia Foundation and NORD last September.
- The "Voice of the Patient" report is available for download now, and a recording of the entire EL-PFDD meeting on galactosemia can be viewed here .
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Saltonstall Unfortunately, today's guidance threatens vital innovation for the thousands of rare diseases that lack FDA-approved therapies.
Key Points:
- Unfortunately, today's guidance threatens vital innovation for the thousands of rare diseases that lack FDA-approved therapies.
- "History tells us without the right incentives, rare disease therapies are not developed, which is why the Orphan Drug Act was created 40 years ago," adds Saltonstall.
- We will also continue to work with the Biden Administration to ensure the Inflation Reduction Act can best support people living with a rare disease," commits Saltonstall.
- While certain aspects of today's guidance are deeply disappointing, NORD is committed to ensuring the Inflation Reduction Act helps meet the complex needs of the more than 25 million Americans living with a rare disease.
