News from NORD

The Morgan Leary Vaughan Fund has received a grant from the Petit Family Foundation in support of the inaugural “Speaking of NEC: Unplugged” event, a one-day regional conference on identifying practical solutions to reduce the devastating effects of necrotizing enterocolitis on premature infants and their families. The event will take place in Hartford, Connecticut, in the spring of 2018.

The Morgan Leary Vaughan Fund has received a grant from the Petit Family Foundation in support of the inaugural “Speaking of NEC: Unplugged” event, a one-day regional conference on identifying practical solutions to reduce the devastating effects of necrotizing enterocolitis on premature infants and their families. The event will take place in Hartford, Connecticut, in the spring of 2018.

The Morgan Leary Vaughan Fund has received a grant from the Petit Family Foundation in support of the inaugural “Speaking of NEC: Unplugged” event, a one-day regional conference on identifying practical solutions to reduce the devastating effects of necrotizing enterocolitis on premature infants and their families. The event will take place in Hartford, Connecticut, in the spring of 2018.

Register today for the first annual STEP UP for SYNGAP1 Walk at the Houston Zoo. Come walk a 5K among the animals and enjoy a day of family fun on Saturday, December 9, 2017.

Register today for the first annual STEP UP for SYNGAP1 Walk at the Houston Zoo. Come walk a 5K among the animals and enjoy a day of family fun on Saturday, December 9, 2017.

Register today for the first annual STEP UP for SYNGAP1 Walk at the Houston Zoo. Come walk a 5K among the animals and enjoy a day of family fun on Saturday, December 9, 2017.

APFED is now accepting applications for three 2018 HOPE Pilot Grants. This program was established to allow investigators from various disciplines to initiate new projects related to eosinophilic-associated diseases. Find additional information here.

Also, join APFED for an evening of music and fundraising in New York City on November 10, 2017. The event will feature Mike DelGuidice and Big Shot, celebrating the music of Billy Joel. More.

APFED is now accepting applications for three 2018 HOPE Pilot Grants. This program was established to allow investigators from various disciplines to initiate new projects related to eosinophilic-associated diseases. Find additional information here.

Also, join APFED for an evening of music and fundraising in New York City on November 10, 2017. The event will feature Mike DelGuidice and Big Shot, celebrating the music of Billy Joel. More.

APFED is now accepting applications for three 2018 HOPE Pilot Grants. This program was established to allow investigators from various disciplines to initiate new projects related to eosinophilic-associated diseases. Find additional information here.

Also, join APFED for an evening of music and fundraising in New York City on November 10, 2017. The event will feature Mike DelGuidice and Big Shot, celebrating the music of Billy Joel. More.

Registration is now open for AIU’s first-ever conference. The event will take place March 23-25, 2018, in Kansas City. Leading experts will speak on a wide variety of topics important to the AI community. More.  

Registration is now open for AIU’s first-ever conference. The event will take place March 23-25, 2018, in Kansas City. Leading experts will speak on a wide variety of topics important to the AI community. More.  

Registration is now open for AIU’s first-ever conference. The event will take place March 23-25, 2018, in Kansas City. Leading experts will speak on a wide variety of topics important to the AI community. More.  

A new documentary video developed by NORD shows how a small community in Kansas came together to support an 11-year-old boy who has a rare disease that makes it necessary for him to avoid sunlight.

Peyton Madden has xeroderma pigmentosum (XP), a rare condition resulting in extreme sensitivity to the sun’s ultraviolet rays. As a result, he is not able to join friends and classmates in typical activities such as swimming in the community pool.

As part of NORD’s “Do Your Share” campaign, community leaders in El Dorado, Kansas, organized a surprise event for Peyton on a recent evening. NORD was on hand with a videographer. Watch the 5-minute video.

A new documentary video developed by NORD shows how a small community in Kansas came together to support an 11-year-old boy who has a rare disease that makes it necessary for him to avoid sunlight.

Peyton Madden has xeroderma pigmentosum (XP), a rare condition resulting in extreme sensitivity to the sun’s ultraviolet rays. As a result, he is not able to join friends and classmates in typical activities such as swimming in the community pool.

As part of NORD’s “Do Your Share” campaign, community leaders in El Dorado, Kansas, organized a surprise event for Peyton on a recent evening. NORD was on hand with a videographer. Watch the 5-minute video.

A new documentary video developed by NORD shows how a small community in Kansas came together to support an 11-year-old boy who has a rare disease that makes it necessary for him to avoid sunlight.

Peyton Madden has xeroderma pigmentosum (XP), a rare condition resulting in extreme sensitivity to the sun’s ultraviolet rays. As a result, he is not able to join friends and classmates in typical activities such as swimming in the community pool.

As part of NORD’s “Do Your Share” campaign, community leaders in El Dorado, Kansas, organized a surprise event for Peyton on a recent evening. NORD was on hand with a videographer. Watch the 5-minute video.

NORD recently joined 140 other patient advocacy organizations in sending a letter to Congress urging that the Orphan Drug Tax Credit remain in place. The Orphan Drug Tax Credit is one of the incentives in the Orphan Drug Act that have promoted R & D on diagnostics and treatments for people with rare diseases since 1983.

As Congress discusses possible approaches to tax reform, some options would include doing away with the ODTC. However, NORD and its policy partners believe patients with rare diseases would suffer if that were to happen. A 2015 study showed that one-third fewer orphan drugs would be developed if the ODTC did not exist.

NORD recently joined 140 other patient advocacy organizations in sending a letter to Congress urging that the Orphan Drug Tax Credit remain in place. The Orphan Drug Tax Credit is one of the incentives in the Orphan Drug Act that have promoted R & D on diagnostics and treatments for people with rare diseases since 1983.

As Congress discusses possible approaches to tax reform, some options would include doing away with the ODTC. However, NORD and its policy partners believe patients with rare diseases would suffer if that were to happen. A 2015 study showed that one-third fewer orphan drugs would be developed if the ODTC did not exist.

NORD recently joined 140 other patient advocacy organizations in sending a letter to Congress urging that the Orphan Drug Tax Credit remain in place. The Orphan Drug Tax Credit is one of the incentives in the Orphan Drug Act that have promoted R & D on diagnostics and treatments for people with rare diseases since 1983.

As Congress discusses possible approaches to tax reform, some options would include doing away with the ODTC. However, NORD and its policy partners believe patients with rare diseases would suffer if that were to happen. A 2015 study showed that one-third fewer orphan drugs would be developed if the ODTC did not exist.

NORD Board Chair Marshall Summar, MD, will moderate a Social Security Administration (SSA) National Disability Forum on Compassionate Allowances and Rare Diseases on Nov. 7, 2017. Speakers will discuss the challenges of living with rare diseases and will recommend specific diseases for SSA’s Compassionate Allowances list.

The Compassionate Allowances list is a list of diseases or medical conditions that almost invariably meet SSA guidelines for disability assistance. Applications from individuals with those diagnoses qualify for expedited review.

Medical professionals and patient representatives are invited to participate in this event. Registration to attend the Washington, DC forum in person or to listen to the forum via teleconference may be done online.

SSA will also be accepting questions or comments about Compassionate Allowances on Twitter during the forum. Use the hashtag #SSANDForum and/or direct comments to @SSAOutreach. Questions or comments may also be sent by email to nationaldisabilityforum@ssa.gov.

For two weeks after the event, SSA will be accepting suggestions online regarding diseases or conditions to be considered for the list. Read about the Compassionate Allowances Initiative and view the current list of conditions here.

NORD Board Chair Marshall Summar, MD, will moderate a Social Security Administration (SSA) National Disability Forum on Compassionate Allowances and Rare Diseases on Nov. 7, 2017. Speakers will discuss the challenges of living with rare diseases and will recommend specific diseases for SSA’s Compassionate Allowances list.

The Compassionate Allowances list is a list of diseases or medical conditions that almost invariably meet SSA guidelines for disability assistance. Applications from individuals with those diagnoses qualify for expedited review.

Medical professionals and patient representatives are invited to participate in this event. Registration to attend the Washington, DC forum in person or to listen to the forum via teleconference may be done online.

SSA will also be accepting questions or comments about Compassionate Allowances on Twitter during the forum. Use the hashtag #SSANDForum and/or direct comments to @SSAOutreach. Questions or comments may also be sent by email to nationaldisabilityforum@ssa.gov.

For two weeks after the event, SSA will be accepting suggestions online regarding diseases or conditions to be considered for the list. Read about the Compassionate Allowances Initiative and view the current list of conditions here.

NORD Board Chair Marshall Summar, MD, will moderate a Social Security Administration (SSA) National Disability Forum on Compassionate Allowances and Rare Diseases on Nov. 7, 2017. Speakers will discuss the challenges of living with rare diseases and will recommend specific diseases for SSA’s Compassionate Allowances list.

The Compassionate Allowances list is a list of diseases or medical conditions that almost invariably meet SSA guidelines for disability assistance. Applications from individuals with those diagnoses qualify for expedited review.

Medical professionals and patient representatives are invited to participate in this event. Registration to attend the Washington, DC forum in person or to listen to the forum via teleconference may be done online.

SSA will also be accepting questions or comments about Compassionate Allowances on Twitter during the forum. Use the hashtag #SSANDForum and/or direct comments to @SSAOutreach. Questions or comments may also be sent by email to nationaldisabilityforum@ssa.gov.

For two weeks after the event, SSA will be accepting suggestions online regarding diseases or conditions to be considered for the list. Read about the Compassionate Allowances Initiative and view the current list of conditions here.

NORD has reopened the call to submit abstracts and letters of intent for the following research grants.

• Cat Eye Syndrome
• Malonic Aciduria
• Post-Orgasmic Illness Syndrome

The deadline is ongoing and all US and international researchers interested in studying these diseases are encouraged to consider applying. For additional information and to view the RFPs, visit the NORD website.

NORD has reopened the call to submit abstracts and letters of intent for the following research grants.

• Cat Eye Syndrome
• Malonic Aciduria
• Post-Orgasmic Illness Syndrome

The deadline is ongoing and all US and international researchers interested in studying these diseases are encouraged to consider applying. For additional information and to view the RFPs, visit the NORD website.

NORD has reopened the call to submit abstracts and letters of intent for the following research grants.

• Cat Eye Syndrome
• Malonic Aciduria
• Post-Orgasmic Illness Syndrome

The deadline is ongoing and all US and international researchers interested in studying these diseases are encouraged to consider applying. For additional information and to view the RFPs, visit the NORD website.

Orphan drugs accounted for only 7.9% of total drug sales in the United States in 2016, according to a new study conducted by the QuintilesIMS Institute. The study, commissioned by NORD, was released at the recent NORD Rare Diseases and Orphan Products Annual Summit in Washington DC.

The study analyzed the role of the Orphan Drug Act, orphan drug usage, and costs. Key findings included that:

• Of the $46 billion spent on pharmaceutical drugs in the US in 2016, only 7.9% was for orphan drugs.
• The orphan drug share of the total volume of pharmaceutical use in the US in 2016 was just 0.3%, down from a peak of 0.6% in 2003.

Findings also suggested that the Orphan Drug Act remains as important today as it was in 1983, when it was enacted. Read the QuintilesIMS report and a corresponding NORD document providing additional analysis.

Orphan drugs accounted for only 7.9% of total drug sales in the United States in 2016, according to a new study conducted by the QuintilesIMS Institute. The study, commissioned by NORD, was released at the recent NORD Rare Diseases and Orphan Products Annual Summit in Washington DC.

The study analyzed the role of the Orphan Drug Act, orphan drug usage, and costs. Key findings included that:

• Of the $46 billion spent on pharmaceutical drugs in the US in 2016, only 7.9% was for orphan drugs.
• The orphan drug share of the total volume of pharmaceutical use in the US in 2016 was just 0.3%, down from a peak of 0.6% in 2003.

Findings also suggested that the Orphan Drug Act remains as important today as it was in 1983, when it was enacted. Read the QuintilesIMS report and a corresponding NORD document providing additional analysis.

Orphan drugs accounted for only 7.9% of total drug sales in the United States in 2016, according to a new study conducted by the QuintilesIMS Institute. The study, commissioned by NORD, was released at the recent NORD Rare Diseases and Orphan Products Annual Summit in Washington DC.

The study analyzed the role of the Orphan Drug Act, orphan drug usage, and costs. Key findings included that:

• Of the $46 billion spent on pharmaceutical drugs in the US in 2016, only 7.9% was for orphan drugs.
• The orphan drug share of the total volume of pharmaceutical use in the US in 2016 was just 0.3%, down from a peak of 0.6% in 2003.

Findings also suggested that the Orphan Drug Act remains as important today as it was in 1983, when it was enacted. Read the QuintilesIMS report and a corresponding NORD document providing additional analysis.

Nominations are now open for the 2018 NORD Rare Impact Awards. These awards honor individuals who have made a positive impact on the rare disease community through research, patient care, advocacy, or other areas of involvement.

Awards are presented in May each year at NORD’s annual Rare Impact Celebration. Nominations may be submitted online. The deadline is January 12, 2018.

In recognition of the 35th anniversaries of NORD and the Orphan Drug Act in 2018, the awards will be presented in four categories representing the four pillars of NORD’s mission: Advocacy, Education, Research, and Patient Assistance.

Honorees from previous years have included members of Congress, staff and senior officials from NIH and FDA, medical researchers and clinicians, patient organization leaders, and individual patients and caregivers. The awards honor those who have helped to improve the lives of those affected by rare diseases.

Nominations are now open for the 2018 NORD Rare Impact Awards. These awards honor individuals who have made a positive impact on the rare disease community through research, patient care, advocacy, or other areas of involvement.

Awards are presented in May each year at NORD’s annual Rare Impact Celebration. Nominations may be submitted online. The deadline is January 12, 2018.

In recognition of the 35th anniversaries of NORD and the Orphan Drug Act in 2018, the awards will be presented in four categories representing the four pillars of NORD’s mission: Advocacy, Education, Research, and Patient Assistance.

Honorees from previous years have included members of Congress, staff and senior officials from NIH and FDA, medical researchers and clinicians, patient organization leaders, and individual patients and caregivers. The awards honor those who have helped to improve the lives of those affected by rare diseases.

Nominations are now open for the 2018 NORD Rare Impact Awards. These awards honor individuals who have made a positive impact on the rare disease community through research, patient care, advocacy, or other areas of involvement.

Awards are presented in May each year at NORD’s annual Rare Impact Celebration. Nominations may be submitted online. The deadline is January 12, 2018.

In recognition of the 35th anniversaries of NORD and the Orphan Drug Act in 2018, the awards will be presented in four categories representing the four pillars of NORD’s mission: Advocacy, Education, Research, and Patient Assistance.

Honorees from previous years have included members of Congress, staff and senior officials from NIH and FDA, medical researchers and clinicians, patient organization leaders, and individual patients and caregivers. The awards honor those who have helped to improve the lives of those affected by rare diseases.