National Organization for Rare Disorders

Last updated

National Organization for Rare Disorders
Founded1983
Legal status 501(c)(3) nonprofit organization
Headquarters55 Kenosia Avenue, Danbury, Connecticut [1]
Servicesprovide support for individuals with rare diseases by advocating and funding research, education, and networking among service providers.
President and CEO
Peter Saltonstall
Website rarediseases.org OOjs UI icon edit-ltr-progressive.svg

The National Organization for Rare Disorders (NORD) is a nonprofit organization, based in Connecticut, [1] aiming to provide support for individuals with rare diseases by advocating and funding research, education, and networking among service providers. [2] It was founded in 1983 by Abbey Meyers, along with individuals and rare diseases leaders of rare disease support groups, and it is a 501(c)(3) tax exempt organization. [2] [3] [4]

Contents

History

The organization grew out of an "informal coalition" of support groups and families called together in the late 1970s to advocate legislation supporting development of orphan drugs, or drugs for treating rare diseases. [5] They succeeded in getting the United States Congress to pass the Orphan Drug Act (ODA) in early 1983. [2] [5] [6]

The initial coalition was led by Abbey Meyers, whose son had Tourette syndrome. [7] Tourette syndrome was estimated by the National Institutes of Health to affect 100,000 people in the United States. Meyers' son was helped by an experimental drug that the manufacturer ceased to develop because they assumed it would not be profitable enough. [4] After passage of the Orphan Drug Act, the coalition founded NORD with Meyers as its president. In 2007 Meyers announced her retirement from the NORD presidency; the president is now Peter L. Saltonstall. [3] [5]

Since its founding in 1983, NORD continued to grow with the help of federal grants and donations. [5]

Activities

NORD's operations include funding research on treatment and cures for rare diseases; lobbying for legislation to benefit the rare diseases community (in addition to the Orphan Drug Act, NORD has helped bring about legislation on publicizing clinical trials on the Internet, to give the public and medical professionals warning about projected drug shortages, and on the development of medical devices); [5] spreading information about rare diseases; and helping individuals with rare diseases afford medication and treatment. [2] [8] In February 2009, NORD sponsored Rare Disease Day in the United States; this was the first time Rare Disease Day was observed in the United States (it was first observed in Europe in February 2008). [9] [10] NORD has also helped other countries to develop orphan drug legislation in their nations.

Organization

Corporate Council

NORD maintains a Corporate Council made up of companies in the pharmaceutical, biotechnology and clinical research industries. [11] Members include:

Partnerships

NORD is engaged in partnerships with a number of patient advocacy groups and industry organizations. Partners include: [12] [13]

See also

Related Research Articles

<span class="mw-page-title-main">Pfizer</span> American multinational pharmaceutical and biotechnology corporation

Pfizer Inc. is an American multinational pharmaceutical and biotechnology corporation headquartered at The Spiral in Manhattan, New York City. The company was established in 1849 in New York by two German entrepreneurs, Charles Pfizer (1824–1906) and his cousin Charles F. Erhart (1821–1891).

A rare disease is a disease that affects a small percentage of the population. In some parts of the world, the term orphan disease describes a rare disease whose rarity results in little or no funding or research for treatments, without financial incentives from governments or other agencies. Orphan drugs are medications targeting orphan diseases.

An orphan drug is a pharmaceutical agent that is developed to treat certain rare medical conditions. An orphan drug would not be profitable to produce without government assistance, due to the small population of patients affected by the conditions. The conditions that orphan drugs are used to treat are referred to as orphan diseases. The assignment of orphan status to a disease and to drugs developed to treat it is a matter of public policy that depends on the legislation of the country.

<span class="mw-page-title-main">Amgen</span> American multinational biopharmaceutical company

Amgen Inc. is an American multinational biopharmaceutical company headquartered in Thousand Oaks, California. One of the world's largest independent biotechnology companies, As of 2022, Amgen has approximately 24,000 staff in total.

<span class="mw-page-title-main">Eisai (company)</span> Japanese pharmaceutical company

Eisai Co., Ltd. is a Japanese pharmaceutical company headquartered in Tokyo, Japan. It has some 10,000 employees, among them about 1,500 in research. Eisai is listed on the Tokyo Stock Exchange and is a member of the Topix 100 and Nikkei 225 stock indices.

BioMarin Pharmaceutical Inc. is an American biotechnology company headquartered in San Rafael, California. It has offices and facilities in the United States, South America, Asia, and Europe. BioMarin's core business and research is in enzyme replacement therapies (ERTs). BioMarin was the first company to provide therapeutics for mucopolysaccharidosis type I, by manufacturing laronidase. BioMarin was also the first company to provide therapeutics for phenylketonuria (PKU).

<span class="mw-page-title-main">PTC Therapeutics</span> Pharmaceutical company

PTC Therapeutics, Inc. is a US pharmaceutical company focused on the development of orally administered small molecule drugs and gene therapy which regulate gene expression by targeting post-transcriptional control (PTC) mechanisms in orphan diseases.

<span class="mw-page-title-main">Sumitomo Pharma</span> Pharmaceutical company based in Japan

Sumitomo Pharma Company Limited is a Japanese multinational pharmaceutical company. The company is focused on oncology, psychiatry, neurology, women's health issues, urological diseases among other areas. Its headquarters are located in Chuo-ku, Osaka.

<span class="mw-page-title-main">Santaris Pharma</span> Biopharmaceutical company founded in Denmark

Santaris Pharma A/S was a biopharmaceutical company founded in 2003 in Copenhagen, Denmark. The company also had a branch in San Diego, California that opened in 2009. Created by a merger between Cureon and Pantheco, Santaris developed RNA-targeted medicines using a Locked Nucleic Acid (LNA) Drug Platform and Drug Development Engine.

<span class="mw-page-title-main">Actelion</span> Swiss biopharmaceutical company

Actelion is a pharmaceuticals and biotechnology company established in December 1997, headquartered in Allschwil near Basel, Switzerland.

<span class="mw-page-title-main">Orphan Drug Act of 1983</span> Law passed in the United States to facilitate development of orphan drugs

The Orphan Drug Act of 1983 is a law passed in the United States to facilitate development of orphan drugs—drugs for rare diseases such as Huntington's disease, myoclonus, ALS, Tourette syndrome or muscular dystrophy which affect small numbers of individuals residing in the United States.

Alexion Pharmaceuticals, a subsidiary of AstraZeneca, is a pharmaceutical company headquartered in Boston, Massachusetts that specializes in orphan drugs to treat rare diseases.

Alnylam Pharmaceuticals, Inc. is an American biopharmaceutical company focused on the discovery, development and commercialization of RNA interference (RNAi) therapeutics for genetically defined diseases. The company was founded in 2002 and is headquartered in Cambridge, Massachusetts. In 2016, Forbes included the company on its "100 Most Innovative Growth Companies" list.

Horizon Therapeutics was a biopharmaceutical company focused on researching, developing, and commercializing medicines that address critical needs for people impacted by rare and rheumatic diseases. Horizon primarily markets products in the United States, which represented 97% of Horizon's 2019 worldwide sales. Amgen acquired the company in October 2023.

<span class="mw-page-title-main">Marathon Pharmaceuticals</span> Former U.S. rare disease drug company

Marathon Pharmaceuticals LLC was a privately held biopharmaceuticals company focused on drugs for people with rare diseases. The Illinois-based company developed and manufactured therapeutics and brought them to market. It employed 100 people in four global locations. In 2017, PTC Therapeutics acquired rights to Marathon Pharmaceuticals' drug Emflaza (deflazacort) for $140 million after criticism about their plan to sell the drug at a list price of $89,000 per year to sufferers despite the fact that the same drug was available in Canada and the UK for around $1,000 per year.

Specialty drugs or specialty pharmaceuticals are a recent designation of pharmaceuticals classified as high-cost, high complexity and/or high touch. Specialty drugs are often biologics—"drugs derived from living cells" that are injectable or infused. They are used to treat complex or rare chronic conditions such as cancer, rheumatoid arthritis, hemophilia, H.I.V. psoriasis, inflammatory bowel disease and hepatitis C. In 1990 there were 10 specialty drugs on the market, around five years later nearly 30, by 2008 200, and by 2015 300.

<span class="mw-page-title-main">Timothy Coté</span> American doctor

Timothy R. Coté is an American doctor, expert of infectious and neoplastic disease, and a former Director of the Office of Orphan Products Development (OOPD) at the Food and Drug Administration (FDA).

CSL Vifor is a global specialty pharmaceuticals company in the treatment areas of iron deficiency, dialysis, nephrology & rare disease. It is headquartered in Switzerland and consists of CSL Vifor, Vifor Fresenius Medical Care Renal Pharma (VFMCRP) and Sanifit Therapeutics.

<span class="mw-page-title-main">Ultragenyx</span> American biopharmaceutical company

Ultragenyx Pharmaceutical Inc. is an American biopharmaceutical company involved in the research and development of novel products for treatment of rare and ultra-rare genetic diseases for which there are typically no approved treatments and high unmet medical need. The company works with multiple drug modalities including biologics, small molecule, gene therapies, and ASO and mRNAs in the disease categories of bone, endocrine, metabolic, muscle and CNS diseases.

References

  1. 1 2 "National Organization for Rare Disorders (NORD)". NIDCD . 23 June 2020. Retrieved 30 September 2023.
  2. 1 2 3 4 "About NORD". National Organization of Rare Diseases. 30 July 2008. Archived from the original on 17 February 2009. Retrieved 14 February 2009.
  3. 1 2 "The National Organization for Rare Disorders (NORD) Names Peter Saltonstall New President". Reuters . 5 May 2008. Archived from the original on 4 January 2013. Retrieved 14 February 2009.
  4. 1 2 "Episode 329: Orphan Drugs". 99% Invisible. 13 November 2018. Retrieved 18 November 2018.
  5. 1 2 3 4 5 Parisse-Brassens, Jerome (July 2007). "Abbey Meyers, President of NORD, announces her retirement". European Organization for Rare Disorders. Archived from the original on 9 October 2007. Retrieved 14 February 2009.
  6. ↑ Henkel, John (1999). "Orphan Drug Law Matures into Medical Mainstay". FDA Consumer . U.S. Food and Drug Administration . Retrieved 14 February 2009.
  7. ↑ Graff, Joyce (2008). "Abbey Meyers and the National Organization for Rare Disorders". Powerful Patient. VHL Family Alliance. Retrieved 14 February 2009.
  8. ↑ Donohue, Paul G. (2 February 2009). "'Rare' diseases aren't rare to those who suffer them". The Standard-Times . Retrieved 14 February 2009.
  9. ↑ "National Organization for Rare Disorders To Partner With Discovery Health and 'MYSTERY DIAGNOSIS' for Rare Disease Day 2009". PR Newswire. 9 February 2009. Archived from the original on 17 June 2009. Retrieved 14 February 2009.
  10. ↑ "February 28th is Rare Disease Day". PharmiWeb.com. 12 February 2009. Archived from the original on 22 February 2014. Retrieved 14 February 2009.
  11. ↑ "Current NORD Corporate Council Members". NORD (National Organization for Rare Disorders). 20 July 2022. Archived from the original on 12 October 2022. Retrieved 12 October 2022.
  12. ↑ "National Partnerships: NORD's Partners and Alliances". NORD (National Organization for Rare Disorders). 5 August 2022. Archived from the original on 12 October 2022. Retrieved 12 October 2022.
  13. ↑ "Partnerships". NORD (National Organization for Rare Disorders). 16 March 2022. Archived from the original on 12 October 2022. Retrieved 12 October 2022.
This page is based on this Wikipedia article
Text is available under the CC BY-SA 4.0 license; additional terms may apply.
Images, videos and audio are available under their respective licenses.