News from NORD

The 2017 Association for Frontotemporal Degeneration (AFTD) Education Conference will take place on May 5 at the Sheraton Inner Harbor Hotel in Baltimore. Online registration will open January 9.

The keynote speaker will be Halima Amjad, MD, MPH, Assistant Professor of Medicine at Johns Hopkins University, whose father was diagnosed with primary progressive aphasia around the time she was graduating from medical school in 2009. Other speakers will include physicians, nurses, and social workers from Johns Hopkins, Duke University, the University of Pennsylvania, and Thomas Jefferson University. Additional information is available on the AFTD website.

The 2017 Association for Frontotemporal Degeneration (AFTD) Education Conference will take place on May 5 at the Sheraton Inner Harbor Hotel in Baltimore. Online registration will open January 9.

The keynote speaker will be Halima Amjad, MD, MPH, Assistant Professor of Medicine at Johns Hopkins University, whose father was diagnosed with primary progressive aphasia around the time she was graduating from medical school in 2009. Other speakers will include physicians, nurses, and social workers from Johns Hopkins, Duke University, the University of Pennsylvania, and Thomas Jefferson University. Additional information is available on the AFTD website.

The 2017 Association for Frontotemporal Degeneration (AFTD) Education Conference will take place on May 5 at the Sheraton Inner Harbor Hotel in Baltimore. Online registration will open January 9.

The keynote speaker will be Halima Amjad, MD, MPH, Assistant Professor of Medicine at Johns Hopkins University, whose father was diagnosed with primary progressive aphasia around the time she was graduating from medical school in 2009. Other speakers will include physicians, nurses, and social workers from Johns Hopkins, Duke University, the University of Pennsylvania, and Thomas Jefferson University. Additional information is available on the AFTD website.

NORD is partnering with researchers at Oregon State University to conduct the first large-scale study of information and psychosocial support needs of people living with rare disorders. The purpose is to assess the needs and find similarities and differences across the spectrum of disorders. An online survey is now open for adults living with rare diseases. The principal investigator is Kathleen Bogart, PhD, Assistant Professor of Psychology at Oregon State. Co-investigator is Veronica Irvin, PhD, MPH, also of OSU.

NORD is partnering with researchers at Oregon State University to conduct the first large-scale study of information and psychosocial support needs of people living with rare disorders. The purpose is to assess the needs and find similarities and differences across the spectrum of disorders. An online survey is now open for adults living with rare diseases. The principal investigator is Kathleen Bogart, PhD, Assistant Professor of Psychology at Oregon State. Co-investigator is Veronica Irvin, PhD, MPH, also of OSU.

NORD is partnering with researchers at Oregon State University to conduct the first large-scale study of information and psychosocial support needs of people living with rare disorders. The purpose is to assess the needs and find similarities and differences across the spectrum of disorders. An online survey is now open for adults living with rare diseases. The principal investigator is Kathleen Bogart, PhD, Assistant Professor of Psychology at Oregon State. Co-investigator is Veronica Irvin, PhD, MPH, also of OSU.

The last day of February is observed in more than 90 countries worldwide as Rare Disease Day to promote better understanding of the more than 7,000 diseases classified as rare. In the US, a disease is considered rare if it is believed to affect fewer than 200,000 Americans. The National Institutes of Health (NIH) lists all known rare diseases on its website.

As the national sponsor of Rare Disease Day in the US, NORD is working with other national alliances around the world to plan a global theme and educational outreach each year. This year’s theme is Research. Events will focus on the importance of research and the need for expanded research on rare diseases.

On the national Rare Disease Day website, which is hosted by NORD, a state-by-state listing of planned events makes it easy to find out what is happening in your state and/or to promote awareness of events you are planning. Many teaching hospitals and academic centers host special programs or lobby tabling events on or around Rare Disease Day.

If you are interested in organizing a program, tabling event, or literature display at your institution, you can download resources from NORD’s Rare Disease Day US website. NORD also can provide speakers for Rare Disease Day (or other) educational events through its Patient/Caregiver Speakers Bureau.  

Medical professionals and students can also show their support for Rare Disease Day by submitting a photo to NORD’s “Handprints Across America” campaign, which will be displayed on the Rare Disease Day US website. Questions related to Rare Disease Day events or requests for speakers or other resources may be directed to education@rarediseases.org.

The last day of February is observed in more than 90 countries worldwide as Rare Disease Day to promote better understanding of the more than 7,000 diseases classified as rare. In the US, a disease is considered rare if it is believed to affect fewer than 200,000 Americans. The National Institutes of Health (NIH) lists all known rare diseases on its website.

As the national sponsor of Rare Disease Day in the US, NORD is working with other national alliances around the world to plan a global theme and educational outreach each year. This year’s theme is Research. Events will focus on the importance of research and the need for expanded research on rare diseases.

On the national Rare Disease Day website, which is hosted by NORD, a state-by-state listing of planned events makes it easy to find out what is happening in your state and/or to promote awareness of events you are planning. Many teaching hospitals and academic centers host special programs or lobby tabling events on or around Rare Disease Day.

If you are interested in organizing a program, tabling event, or literature display at your institution, you can download resources from NORD’s Rare Disease Day US website. NORD also can provide speakers for Rare Disease Day (or other) educational events through its Patient/Caregiver Speakers Bureau.  

Medical professionals and students can also show their support for Rare Disease Day by submitting a photo to NORD’s “Handprints Across America” campaign, which will be displayed on the Rare Disease Day US website. Questions related to Rare Disease Day events or requests for speakers or other resources may be directed to education@rarediseases.org.

The last day of February is observed in more than 90 countries worldwide as Rare Disease Day to promote better understanding of the more than 7,000 diseases classified as rare. In the US, a disease is considered rare if it is believed to affect fewer than 200,000 Americans. The National Institutes of Health (NIH) lists all known rare diseases on its website.

As the national sponsor of Rare Disease Day in the US, NORD is working with other national alliances around the world to plan a global theme and educational outreach each year. This year’s theme is Research. Events will focus on the importance of research and the need for expanded research on rare diseases.

On the national Rare Disease Day website, which is hosted by NORD, a state-by-state listing of planned events makes it easy to find out what is happening in your state and/or to promote awareness of events you are planning. Many teaching hospitals and academic centers host special programs or lobby tabling events on or around Rare Disease Day.

If you are interested in organizing a program, tabling event, or literature display at your institution, you can download resources from NORD’s Rare Disease Day US website. NORD also can provide speakers for Rare Disease Day (or other) educational events through its Patient/Caregiver Speakers Bureau.  

Medical professionals and students can also show their support for Rare Disease Day by submitting a photo to NORD’s “Handprints Across America” campaign, which will be displayed on the Rare Disease Day US website. Questions related to Rare Disease Day events or requests for speakers or other resources may be directed to education@rarediseases.org.

The Spinal CSF Leak Foundation has updated its resource documents for medical professionals and patients. The newly revised materials are available for free download online. Topics include an overview of this condition, diagnosis, causes, treatment, and classification of spontaneous spinal CSF leaks.

The Spinal CSF Leak Foundation has updated its resource documents for medical professionals and patients. The newly revised materials are available for free download online. Topics include an overview of this condition, diagnosis, causes, treatment, and classification of spontaneous spinal CSF leaks.

The Spinal CSF Leak Foundation has updated its resource documents for medical professionals and patients. The newly revised materials are available for free download online. Topics include an overview of this condition, diagnosis, causes, treatment, and classification of spontaneous spinal CSF leaks.

A majority of states are not effectively addressing the challenges of living with rare diseases through state legislation and public health policies, a new NORD report shows. NORD’s State Report Card: A Roadmap to Improve the Lives of People with Rare Diseases measures states’ effectiveness in specific policy areas such as newborn screening, coverage for medical foods, biosimilar prescriber communications, protection against step therapy protocols, and establishment of rare disease advisory councils.

At the time the report was released, NORD also launched a new Rare Action Network website to help advocates address specific rare disease issues within their states.

A majority of states are not effectively addressing the challenges of living with rare diseases through state legislation and public health policies, a new NORD report shows. NORD’s State Report Card: A Roadmap to Improve the Lives of People with Rare Diseases measures states’ effectiveness in specific policy areas such as newborn screening, coverage for medical foods, biosimilar prescriber communications, protection against step therapy protocols, and establishment of rare disease advisory councils.

At the time the report was released, NORD also launched a new Rare Action Network website to help advocates address specific rare disease issues within their states.

A majority of states are not effectively addressing the challenges of living with rare diseases through state legislation and public health policies, a new NORD report shows. NORD’s State Report Card: A Roadmap to Improve the Lives of People with Rare Diseases measures states’ effectiveness in specific policy areas such as newborn screening, coverage for medical foods, biosimilar prescriber communications, protection against step therapy protocols, and establishment of rare disease advisory councils.

At the time the report was released, NORD also launched a new Rare Action Network website to help advocates address specific rare disease issues within their states.

Do you have a patient or colleague who has demonstrated extraordinary commitment to advancing understanding of rare diseases or improving the lives of those affected by rare diseases? January 13th is the deadline to nominate individuals for NORD’s Rare Impact Awards. Nominations can be submitted through the NORD website.

NORD’s Rare Impact Awards ceremony will take place on May 18, 2017, in the amphitheater of the Ronald Reagan Building and International Trade Center, the largest structure in Washington, DC, and the first and only federal building dedicated to both federal and private use. More than 500 distinguished guests are expected to attend. Registration will be available soon on the NORD website. 

The Rare Impact Awards honor individuals and organizations for commitment to improving the lives of patients and families affected by rare diseases. Nominees may include patients, caregivers, clinicians, researchers, advocates, and others who in some way have contributed to the greater good of the community. Nominations are also being sought for organizations that have helped drive better understanding of rare diseases and/or improved care for those affected.

Do you have a patient or colleague who has demonstrated extraordinary commitment to advancing understanding of rare diseases or improving the lives of those affected by rare diseases? January 13th is the deadline to nominate individuals for NORD’s Rare Impact Awards. Nominations can be submitted through the NORD website.

NORD’s Rare Impact Awards ceremony will take place on May 18, 2017, in the amphitheater of the Ronald Reagan Building and International Trade Center, the largest structure in Washington, DC, and the first and only federal building dedicated to both federal and private use. More than 500 distinguished guests are expected to attend. Registration will be available soon on the NORD website. 

The Rare Impact Awards honor individuals and organizations for commitment to improving the lives of patients and families affected by rare diseases. Nominees may include patients, caregivers, clinicians, researchers, advocates, and others who in some way have contributed to the greater good of the community. Nominations are also being sought for organizations that have helped drive better understanding of rare diseases and/or improved care for those affected.

Do you have a patient or colleague who has demonstrated extraordinary commitment to advancing understanding of rare diseases or improving the lives of those affected by rare diseases? January 13th is the deadline to nominate individuals for NORD’s Rare Impact Awards. Nominations can be submitted through the NORD website.

NORD’s Rare Impact Awards ceremony will take place on May 18, 2017, in the amphitheater of the Ronald Reagan Building and International Trade Center, the largest structure in Washington, DC, and the first and only federal building dedicated to both federal and private use. More than 500 distinguished guests are expected to attend. Registration will be available soon on the NORD website. 

The Rare Impact Awards honor individuals and organizations for commitment to improving the lives of patients and families affected by rare diseases. Nominees may include patients, caregivers, clinicians, researchers, advocates, and others who in some way have contributed to the greater good of the community. Nominations are also being sought for organizations that have helped drive better understanding of rare diseases and/or improved care for those affected.

Hackensack University Medical Center has been designated a center of excellence by the Hereditary Neuropathy Foundation. The designation was made as a result of the medical center’s clinical care, community engagement, research, and educate related to Charcot-Marie-Tooth disease/hereditary neuropathies.

Hackensack University Medical Center has been designated a center of excellence by the Hereditary Neuropathy Foundation. The designation was made as a result of the medical center’s clinical care, community engagement, research, and educate related to Charcot-Marie-Tooth disease/hereditary neuropathies.

Hackensack University Medical Center has been designated a center of excellence by the Hereditary Neuropathy Foundation. The designation was made as a result of the medical center’s clinical care, community engagement, research, and educate related to Charcot-Marie-Tooth disease/hereditary neuropathies.

The disease formerly known as primary biliary cirrhosis now is called primary biliary cholangitis (PBC), and PSC Partners Seeking a Cure has launched a campaign to promote awareness of the difference between that condition and primary sclerosing cholangitis (PSC). Despite sharing certain similar characteristics, the two diseases are not the same and require different treatments and monitoring. Learn more on the PSC Partners website.

The disease formerly known as primary biliary cirrhosis now is called primary biliary cholangitis (PBC), and PSC Partners Seeking a Cure has launched a campaign to promote awareness of the difference between that condition and primary sclerosing cholangitis (PSC). Despite sharing certain similar characteristics, the two diseases are not the same and require different treatments and monitoring. Learn more on the PSC Partners website.

The disease formerly known as primary biliary cirrhosis now is called primary biliary cholangitis (PBC), and PSC Partners Seeking a Cure has launched a campaign to promote awareness of the difference between that condition and primary sclerosing cholangitis (PSC). Despite sharing certain similar characteristics, the two diseases are not the same and require different treatments and monitoring. Learn more on the PSC Partners website.

New guidelines for the diagnosis and management of cystathionine beta-synthase deficiency have been published in the Journal of Inherited Metabolic Disease. The HCU Network Australia recently hosted an online webinar featuring Andrew Morris MD, the primary author of the new guidelines.

New guidelines for the diagnosis and management of cystathionine beta-synthase deficiency have been published in the Journal of Inherited Metabolic Disease. The HCU Network Australia recently hosted an online webinar featuring Andrew Morris MD, the primary author of the new guidelines.

New guidelines for the diagnosis and management of cystathionine beta-synthase deficiency have been published in the Journal of Inherited Metabolic Disease. The HCU Network Australia recently hosted an online webinar featuring Andrew Morris MD, the primary author of the new guidelines.

Each year the National Organization for Rare Disorders (NORD) hosts its Rare Impact Awards celebration in May. At this event, individuals and organizations are honored for advancing progress for patients and families affected by rare diseases. Nominees can include physicians, researchers, patients, caregivers, advocates, and others. In addition, organizations are honored for outstanding service to the rare disease community.

Honorees in recent years have included Francis S. Collins, MD, PhD, Director of the National Institutes of Health; Margaret A. Hamburg, MD, former Commissioner of the Food and Drug Administration; Stephen Cederbaum, MD, of the University of California, Los Angeles; and Arthur Caplan, PhD, of the NYU Langone Medical Center.

The 2017 Rare Impact Awards celebration will take place on May 18 in Washington, DC. Visit the NORD website for additional information and to submit nominations.

Each year the National Organization for Rare Disorders (NORD) hosts its Rare Impact Awards celebration in May. At this event, individuals and organizations are honored for advancing progress for patients and families affected by rare diseases. Nominees can include physicians, researchers, patients, caregivers, advocates, and others. In addition, organizations are honored for outstanding service to the rare disease community.

Honorees in recent years have included Francis S. Collins, MD, PhD, Director of the National Institutes of Health; Margaret A. Hamburg, MD, former Commissioner of the Food and Drug Administration; Stephen Cederbaum, MD, of the University of California, Los Angeles; and Arthur Caplan, PhD, of the NYU Langone Medical Center.

The 2017 Rare Impact Awards celebration will take place on May 18 in Washington, DC. Visit the NORD website for additional information and to submit nominations.

Each year the National Organization for Rare Disorders (NORD) hosts its Rare Impact Awards celebration in May. At this event, individuals and organizations are honored for advancing progress for patients and families affected by rare diseases. Nominees can include physicians, researchers, patients, caregivers, advocates, and others. In addition, organizations are honored for outstanding service to the rare disease community.

Honorees in recent years have included Francis S. Collins, MD, PhD, Director of the National Institutes of Health; Margaret A. Hamburg, MD, former Commissioner of the Food and Drug Administration; Stephen Cederbaum, MD, of the University of California, Los Angeles; and Arthur Caplan, PhD, of the NYU Langone Medical Center.

The 2017 Rare Impact Awards celebration will take place on May 18 in Washington, DC. Visit the NORD website for additional information and to submit nominations.