User login
We extend our sincere thanks to busy health care professionals for taking time to engage with this issue, read the latest advances, and provide the best possible care for your patients. Your dedication is an inspiration, and we value the impact you make in the lives of others.
The year 2023 marks the 40th anniversary of the Orphan Drug Act (ODA), landmark legislation that incentivized drug companies to dedicate more resources towards the development of therapies for people with rare conditions. At the same time, we celebrate NORD’s 40th anniversary. The coalition of rare disease advocates who sparked rare disease advocacy and convinced lawmakers to pass the ODA in 1983 established NORD that same year to provide an ongoing, unified voice for the needs of the rare disease community. For 4 decades, NORD has worked tirelessly to drive supportive policies, advance medical research, and provide education and services for the now 30 million Americans with a rare disease, of which half are children.
In this issue of the Rare Neurological Disease Special Report, you will learn more about the history of the Orphan Drug Act and the founding of NORD. You will also find articles from rare disease specialists on specific diseases and some of the newest therapies approved under the ODA for conditions such as Friedreich ataxia, Fabry disease, Duchenne muscular dystrophy, and amyotrophic lateral sclerosis. Also in the issue are articles about stiff person syndrome and Guillain-Barré syndrome and COVID vaccination.
We invite you to visit NORD’s webpage (www.rarediseases.org) to access resources for yourself and the patients and families you serve. NORD’s Continuing Medical Education Video Library offers cost-free, for-credit, on-demand rare disease courses developed in collaboration with Platform Q Health. NORD’s Caring for Rare quarterly newsletter provides updates on educational opportunities, events, and issues important for the rare disease community. NORD’s Rare Disease Database provides expert-reviewed reports on rare diseases in patient-friendly language.
As we celebrate the incredible progress made over the past 40 years, we also recognize that more than 95% of rare conditions still lack effective therapies. Continued research, development of new orphan products, and advances in treatment and care are needed. NORD will remain steadfast in its commitment to driving progress, inspiring innovation, and providing services for the rare community. We are deeply appreciative of the support you provide to people living with neurological conditions and encourage you to contact NORD any time we can be helpful to you.
Edward Neilan, MD, PhD
NORD’s Chief Medical Officer
We extend our sincere thanks to busy health care professionals for taking time to engage with this issue, read the latest advances, and provide the best possible care for your patients. Your dedication is an inspiration, and we value the impact you make in the lives of others.
The year 2023 marks the 40th anniversary of the Orphan Drug Act (ODA), landmark legislation that incentivized drug companies to dedicate more resources towards the development of therapies for people with rare conditions. At the same time, we celebrate NORD’s 40th anniversary. The coalition of rare disease advocates who sparked rare disease advocacy and convinced lawmakers to pass the ODA in 1983 established NORD that same year to provide an ongoing, unified voice for the needs of the rare disease community. For 4 decades, NORD has worked tirelessly to drive supportive policies, advance medical research, and provide education and services for the now 30 million Americans with a rare disease, of which half are children.
In this issue of the Rare Neurological Disease Special Report, you will learn more about the history of the Orphan Drug Act and the founding of NORD. You will also find articles from rare disease specialists on specific diseases and some of the newest therapies approved under the ODA for conditions such as Friedreich ataxia, Fabry disease, Duchenne muscular dystrophy, and amyotrophic lateral sclerosis. Also in the issue are articles about stiff person syndrome and Guillain-Barré syndrome and COVID vaccination.
We invite you to visit NORD’s webpage (www.rarediseases.org) to access resources for yourself and the patients and families you serve. NORD’s Continuing Medical Education Video Library offers cost-free, for-credit, on-demand rare disease courses developed in collaboration with Platform Q Health. NORD’s Caring for Rare quarterly newsletter provides updates on educational opportunities, events, and issues important for the rare disease community. NORD’s Rare Disease Database provides expert-reviewed reports on rare diseases in patient-friendly language.
As we celebrate the incredible progress made over the past 40 years, we also recognize that more than 95% of rare conditions still lack effective therapies. Continued research, development of new orphan products, and advances in treatment and care are needed. NORD will remain steadfast in its commitment to driving progress, inspiring innovation, and providing services for the rare community. We are deeply appreciative of the support you provide to people living with neurological conditions and encourage you to contact NORD any time we can be helpful to you.
Edward Neilan, MD, PhD
NORD’s Chief Medical Officer
We extend our sincere thanks to busy health care professionals for taking time to engage with this issue, read the latest advances, and provide the best possible care for your patients. Your dedication is an inspiration, and we value the impact you make in the lives of others.
The year 2023 marks the 40th anniversary of the Orphan Drug Act (ODA), landmark legislation that incentivized drug companies to dedicate more resources towards the development of therapies for people with rare conditions. At the same time, we celebrate NORD’s 40th anniversary. The coalition of rare disease advocates who sparked rare disease advocacy and convinced lawmakers to pass the ODA in 1983 established NORD that same year to provide an ongoing, unified voice for the needs of the rare disease community. For 4 decades, NORD has worked tirelessly to drive supportive policies, advance medical research, and provide education and services for the now 30 million Americans with a rare disease, of which half are children.
In this issue of the Rare Neurological Disease Special Report, you will learn more about the history of the Orphan Drug Act and the founding of NORD. You will also find articles from rare disease specialists on specific diseases and some of the newest therapies approved under the ODA for conditions such as Friedreich ataxia, Fabry disease, Duchenne muscular dystrophy, and amyotrophic lateral sclerosis. Also in the issue are articles about stiff person syndrome and Guillain-Barré syndrome and COVID vaccination.
We invite you to visit NORD’s webpage (www.rarediseases.org) to access resources for yourself and the patients and families you serve. NORD’s Continuing Medical Education Video Library offers cost-free, for-credit, on-demand rare disease courses developed in collaboration with Platform Q Health. NORD’s Caring for Rare quarterly newsletter provides updates on educational opportunities, events, and issues important for the rare disease community. NORD’s Rare Disease Database provides expert-reviewed reports on rare diseases in patient-friendly language.
As we celebrate the incredible progress made over the past 40 years, we also recognize that more than 95% of rare conditions still lack effective therapies. Continued research, development of new orphan products, and advances in treatment and care are needed. NORD will remain steadfast in its commitment to driving progress, inspiring innovation, and providing services for the rare community. We are deeply appreciative of the support you provide to people living with neurological conditions and encourage you to contact NORD any time we can be helpful to you.
Edward Neilan, MD, PhD
NORD’s Chief Medical Officer
<!--$RCSfile: InCopy_agile.xsl,v $ $Revision: 1.35 $-->
<!--$RCSfile: drupal.xsl,v $ $Revision: 1.7 $-->
<root generator="drupal.xsl" gversion="1.7"> <header> <fileName>165335_web2</fileName> <TBEID>0C04C7D5.SIG</TBEID> <TBUniqueIdentifier>MD_0C04C7D5</TBUniqueIdentifier> <newsOrJournal>News</newsOrJournal> <publisherName>Frontline Medical Communications</publisherName> <storyname/> <articleType>2</articleType> <TBLocation>Hold-NR SUPPLEMENTS</TBLocation> <QCDate/> <firstPublished>20231010T152958</firstPublished> <LastPublished>20231010T152958</LastPublished> <pubStatus qcode="stat:"/> <embargoDate>20231013T000100</embargoDate> <killDate/> <CMSDate>20231013T000100</CMSDate> <articleSource/> <facebookInfo/> <meetingNumber/> <byline/> <bylineText/> <bylineFull/> <bylineTitleText/> <USOrGlobal/> <wireDocType/> <newsDocType/> <journalDocType/> <linkLabel/> <pageRange/> <citation/> <quizID/> <indexIssueDate/> <itemClass qcode="ninat:text"/> <provider qcode="provider:imng"> <name>IMNG Medical Media</name> <rightsInfo> <copyrightHolder> <name>Frontline Medical News</name> </copyrightHolder> <copyrightNotice>Copyright (c) 2015 Frontline Medical News, a Frontline Medical Communications Inc. company. All rights reserved. This material may not be published, broadcast, copied, or otherwise reproduced or distributed without the prior written permission of Frontline Medical Communications Inc.</copyrightNotice> </rightsInfo> </provider> <abstract/> <metaDescription>The National Organization for Rare Disorders (NORD) is pleased to partner with Neurology Reviews to provide timely updates on rare disease research, diagnosis, </metaDescription> <articlePDF/> <teaserImage>297661</teaserImage> <teaser>In this issue of the Rare Neurological Disease Special Report, you will learn more about the history of the Orphan Drug Act and the founding of NORD. </teaser> <title>A note from NORD</title> <deck/> <disclaimer/> <AuthorList/> <articleURL/> <doi/> <pubMedID/> <publishXMLStatus/> <publishXMLVersion>1</publishXMLVersion> <useEISSN>0</useEISSN> <urgency/> <pubPubdateYear/> <pubPubdateMonth/> <pubPubdateDay/> <pubVolume/> <pubNumber/> <wireChannels/> <primaryCMSID/> <CMSIDs/> <keywords/> <seeAlsos/> <publications_g> <publicationData> <publicationCode>nr</publicationCode> <pubIssueName/> <pubArticleType/> <pubTopics/> <pubCategories/> <pubSections/> <journalTitle>Neurology Reviews</journalTitle> <journalFullTitle>Neurology Reviews</journalFullTitle> <copyrightStatement>2018 Frontline Medical Communications Inc.,</copyrightStatement> </publicationData> </publications_g> <publications> <term canonical="true">22</term> </publications> <sections> <term canonical="true">75329</term> </sections> <topics> <term canonical="true">27442</term> </topics> <links> <link> <itemClass qcode="ninat:picture"/> <altRep contenttype="image/jpeg">images/24012228.jpg</altRep> <description role="drol:caption">Dr. Edward Neilan</description> <description role="drol:credit"/> </link> </links> </header> <itemSet> <newsItem> <itemMeta> <itemRole>Main</itemRole> <itemClass>text</itemClass> <title>A note from NORD</title> <deck/> </itemMeta> <itemContent> <p><span class="tag metaDescription">The National Organization for Rare Disorders (NORD) is pleased to partner with Neurology Reviews to provide timely updates on rare disease research, diagnosis, and treatments.</span> We extend our sincere thanks to busy health care professionals for taking time to engage with this issue, read the latest advances, and provide the best possible care for your patients. Your dedication is an inspiration, and we value the impact you make in the lives of others. </p> <p>[[{"fid":"297661","view_mode":"medstat_image_flush_right","fields":{"format":"medstat_image_flush_right","field_file_image_alt_text[und][0][value]":"Edward Neilan, chief medical officer, National Organization of Rare Disorders (NORD)","field_file_image_credit[und][0][value]":"","field_file_image_caption[und][0][value]":"Dr. Edward Neilan"},"type":"media","attributes":{"class":"media-element file-medstat_image_flush_right"}}]]The year 2023 marks the 40th anniversary of the Orphan Drug Act (ODA), landmark legislation that incentivized drug companies to dedicate more resources towards the development of therapies for people with rare conditions. At the same time, we celebrate NORD’s 40th anniversary. The coalition of rare disease advocates who sparked rare disease advocacy and convinced lawmakers to pass the ODA in 1983 established NORD that same year to provide an ongoing, unified voice for the needs of the rare disease community. For 4 decades, NORD has worked tirelessly to drive supportive policies, advance medical research, and provide education and services for the now 30 million Americans with a rare disease, of which half are children.<br/><br/>In this issue of the Rare Neurological Disease Special Report, you will learn more about the history of the Orphan Drug Act and the founding of NORD. You will also find articles from rare disease specialists on specific diseases and some of the newest therapies approved under the ODA for conditions such as Friedreich ataxia, Fabry disease, Duchenne muscular dystrophy, and amyotrophic lateral sclerosis. Also in the issue are articles about stiff person syndrome and Guillain-Barré syndrome and COVID vaccination. <br/><br/>We invite you to visit NORD’s webpage (<span class="Hyperlink"><a href="http://www.rarediseases.org">www.rarediseases.org</a></span>) to access resources for yourself and the patients and families you serve. NORD’s Continuing Medical Education Video Library offers cost-free, for-credit, on-demand rare disease courses developed in collaboration with Platform Q Health. NORD’s Caring for Rare quarterly newsletter provides updates on educational opportunities, events, and issues important for the rare disease community. NORD’s Rare Disease Database provides expert-reviewed reports on rare diseases in patient-friendly language. <br/><br/>As we celebrate the incredible progress made over the past 40 years, we also recognize that more than 95% of rare conditions still lack effective therapies. Continued research, development of new orphan products, and advances in treatment and care are needed. NORD will remain steadfast in its commitment to driving progress, inspiring innovation, and providing services for the rare community. We are deeply appreciative of the support you provide to people living with neurological conditions and encourage you to contact NORD any time we can be helpful to you. <br/><br/><em><br/><br/>Edward Neilan, MD, PhD <br/><br/>NORD’s Chief Medical Officer</em></p> </itemContent> </newsItem> <newsItem> <itemMeta> <itemRole>teaser</itemRole> <itemClass>text</itemClass> <title/> <deck/> </itemMeta> <itemContent> </itemContent> </newsItem> </itemSet></root>
