News from NORD

On Rare Disease Day 2017 (Feb. 28th) NORD launched a year-long public awareness campaign featuring the voices and stories of children and adults living with rare diseases. The purpose is to promote greater public awareness. The campaign includes a public service announcement and supporting website where those living with rare diseases may share their stories.

On Rare Disease Day 2017 (Feb. 28th) NORD launched a year-long public awareness campaign featuring the voices and stories of children and adults living with rare diseases. The purpose is to promote greater public awareness. The campaign includes a public service announcement and supporting website where those living with rare diseases may share their stories.

On Rare Disease Day 2017 (Feb. 28th) NORD launched a year-long public awareness campaign featuring the voices and stories of children and adults living with rare diseases. The purpose is to promote greater public awareness. The campaign includes a public service announcement and supporting website where those living with rare diseases may share their stories.

In its efforts to protect health care coverage of patients with rare diseases, NORD has submitted a letter to Congress emphasizing the importance of health insurance protection for patients with rare diseases in any legislation drafted to replace the Affordable Care Act.

In its efforts to protect health care coverage of patients with rare diseases, NORD has submitted a letter to Congress emphasizing the importance of health insurance protection for patients with rare diseases in any legislation drafted to replace the Affordable Care Act.

In its efforts to protect health care coverage of patients with rare diseases, NORD has submitted a letter to Congress emphasizing the importance of health insurance protection for patients with rare diseases in any legislation drafted to replace the Affordable Care Act.

Before the Affordable Care Act was enacted in 2010, many patients with rare diseases could not access health care coverage due to various discriminatory insurance practices, limited Medicaid eligibility, and debilitating cost-sharing. As the administration and Congress discuss repealing and/or replacing the ACA, NORD has published a Principles for Health Coverage Reform document outlining basic principles that must be maintained to protect health care coverage for rare disease patients.

Before the Affordable Care Act was enacted in 2010, many patients with rare diseases could not access health care coverage due to various discriminatory insurance practices, limited Medicaid eligibility, and debilitating cost-sharing. As the administration and Congress discuss repealing and/or replacing the ACA, NORD has published a Principles for Health Coverage Reform document outlining basic principles that must be maintained to protect health care coverage for rare disease patients.

Before the Affordable Care Act was enacted in 2010, many patients with rare diseases could not access health care coverage due to various discriminatory insurance practices, limited Medicaid eligibility, and debilitating cost-sharing. As the administration and Congress discuss repealing and/or replacing the ACA, NORD has published a Principles for Health Coverage Reform document outlining basic principles that must be maintained to protect health care coverage for rare disease patients.

The National Organization for Rare Disorders (NORD) has awarded seven new research grants to fund rare disease research. These grants are in response to NORD’s 2016 Requests for Proposals. The 2017 Requests for Proposals will be published soon on the NORD website, where funding opportunities from NORD member organizations also are posted.

The new grant recipients are:

For the study of Alveolar Capillary Dysplasia with Misalignment of the Pulmonary Veins (ACD/MPV), with support funding raised by the David Ashwell Foundation and the Alveolar Capillary Dysplasia Association

• Przemyslaw Szafranski PhD, Baylor College of Medicine

For the study of Appendix Cancer and Pseudomyxoma Peritonei (PMP), with support funding raised by the Appendix Cancer Pseudomyxoma Peritonei Research Foundation

• Venkatesh Govindarajan PhD, Creighton University
• David L. Morris MD PhD, St. George Hospital, Australia

For the study of Autoimmune Polyglandular Syndrome Type 1 (APS Type 1), with support funding raised by the APS Type 1 Foundation

• Maureen A. Su MD, University of North Carolina at Chapel Hill

For the study of Homocystinuria due to Cystathionine Beta-Synthase Deficiency, with support funding raised from public donations

• Warren D. Kruger PhD, The Research Institute of Fox Chase Cancer Center

For the study of Malonic Aciduria, with support funding raised by the Hope Fund, Lundbeck “Raise Your Hand” campaign, and public donations

• Michael J. Wolfgang PhD, Johns Hopkins University School of Medicine

For the study of Stiff Person Syndrome, with support funding raised by the Lundbeck “Raise Your Hand” campaign and public donations

• Sarah Crisp PhD, University College, London

Grants are made possible by donations to NORD research funds and the assistance of rare disease medical experts who serve on NORD’s Medical Advisory Committee.

The National Organization for Rare Disorders (NORD) has awarded seven new research grants to fund rare disease research. These grants are in response to NORD’s 2016 Requests for Proposals. The 2017 Requests for Proposals will be published soon on the NORD website, where funding opportunities from NORD member organizations also are posted.

The new grant recipients are:

For the study of Alveolar Capillary Dysplasia with Misalignment of the Pulmonary Veins (ACD/MPV), with support funding raised by the David Ashwell Foundation and the Alveolar Capillary Dysplasia Association

• Przemyslaw Szafranski PhD, Baylor College of Medicine

For the study of Appendix Cancer and Pseudomyxoma Peritonei (PMP), with support funding raised by the Appendix Cancer Pseudomyxoma Peritonei Research Foundation

• Venkatesh Govindarajan PhD, Creighton University
• David L. Morris MD PhD, St. George Hospital, Australia

For the study of Autoimmune Polyglandular Syndrome Type 1 (APS Type 1), with support funding raised by the APS Type 1 Foundation

• Maureen A. Su MD, University of North Carolina at Chapel Hill

For the study of Homocystinuria due to Cystathionine Beta-Synthase Deficiency, with support funding raised from public donations

• Warren D. Kruger PhD, The Research Institute of Fox Chase Cancer Center

For the study of Malonic Aciduria, with support funding raised by the Hope Fund, Lundbeck “Raise Your Hand” campaign, and public donations

• Michael J. Wolfgang PhD, Johns Hopkins University School of Medicine

For the study of Stiff Person Syndrome, with support funding raised by the Lundbeck “Raise Your Hand” campaign and public donations

• Sarah Crisp PhD, University College, London

Grants are made possible by donations to NORD research funds and the assistance of rare disease medical experts who serve on NORD’s Medical Advisory Committee.

The National Organization for Rare Disorders (NORD) has awarded seven new research grants to fund rare disease research. These grants are in response to NORD’s 2016 Requests for Proposals. The 2017 Requests for Proposals will be published soon on the NORD website, where funding opportunities from NORD member organizations also are posted.

The new grant recipients are:

For the study of Alveolar Capillary Dysplasia with Misalignment of the Pulmonary Veins (ACD/MPV), with support funding raised by the David Ashwell Foundation and the Alveolar Capillary Dysplasia Association

• Przemyslaw Szafranski PhD, Baylor College of Medicine

For the study of Appendix Cancer and Pseudomyxoma Peritonei (PMP), with support funding raised by the Appendix Cancer Pseudomyxoma Peritonei Research Foundation

• Venkatesh Govindarajan PhD, Creighton University
• David L. Morris MD PhD, St. George Hospital, Australia

For the study of Autoimmune Polyglandular Syndrome Type 1 (APS Type 1), with support funding raised by the APS Type 1 Foundation

• Maureen A. Su MD, University of North Carolina at Chapel Hill

For the study of Homocystinuria due to Cystathionine Beta-Synthase Deficiency, with support funding raised from public donations

• Warren D. Kruger PhD, The Research Institute of Fox Chase Cancer Center

For the study of Malonic Aciduria, with support funding raised by the Hope Fund, Lundbeck “Raise Your Hand” campaign, and public donations

• Michael J. Wolfgang PhD, Johns Hopkins University School of Medicine

For the study of Stiff Person Syndrome, with support funding raised by the Lundbeck “Raise Your Hand” campaign and public donations

• Sarah Crisp PhD, University College, London

Grants are made possible by donations to NORD research funds and the assistance of rare disease medical experts who serve on NORD’s Medical Advisory Committee.

101 Practical Tips for Dealing with CMT is the title of a new book published by the Charcot-Marie-Tooth Association. Written by members of the CMT community, the book contains information for anyone dealing with CMT or muscular dystrophy.

101 Practical Tips for Dealing with CMT is the title of a new book published by the Charcot-Marie-Tooth Association. Written by members of the CMT community, the book contains information for anyone dealing with CMT or muscular dystrophy.

101 Practical Tips for Dealing with CMT is the title of a new book published by the Charcot-Marie-Tooth Association. Written by members of the CMT community, the book contains information for anyone dealing with CMT or muscular dystrophy.

Before the Affordable Care Act was enacted in 2010, many patients with rare diseases could not access health care coverage due to various discriminatory insurance practices, limited Medicaid eligibility, and debilitating cost-sharing. As the administration and Congress discuss repealing and/or replacing the ACA, NORD has published a Principles for Health Coverage Reform document outlining basic principles that must be maintained to protect health care coverage for rare disease patients.

Before the Affordable Care Act was enacted in 2010, many patients with rare diseases could not access health care coverage due to various discriminatory insurance practices, limited Medicaid eligibility, and debilitating cost-sharing. As the administration and Congress discuss repealing and/or replacing the ACA, NORD has published a Principles for Health Coverage Reform document outlining basic principles that must be maintained to protect health care coverage for rare disease patients.

Before the Affordable Care Act was enacted in 2010, many patients with rare diseases could not access health care coverage due to various discriminatory insurance practices, limited Medicaid eligibility, and debilitating cost-sharing. As the administration and Congress discuss repealing and/or replacing the ACA, NORD has published a Principles for Health Coverage Reform document outlining basic principles that must be maintained to protect health care coverage for rare disease patients.

The National Organization for Rare Disorders (NORD) has awarded seven new research grants to fund rare disease research. These grants are in response to NORD’s 2016 Requests for Proposals. The 2017 Requests for Proposals will be published soon on the NORD website, where funding opportunities from NORD member organizations also are posted.

The new grant recipients are:

For the study of Alveolar Capillary Dysplasia with Misalignment of the Pulmonary Veins (ACD/MPV), with support funding raised by the David Ashwell Foundation and the Alveolar Capillary Dysplasia Association

• Przemyslaw Szafranski PhD, Baylor College of Medicine

For the study of Appendix Cancer and Pseudomyxoma Peritonei (PMP), with support funding raised by the Appendix Cancer Pseudomyxoma Peritonei Research Foundation

• Venkatesh Govindarajan PhD, Creighton University
• David L. Morris MD PhD, St. George Hospital, Australia

For the study of Autoimmune Polyglandular Syndrome Type 1 (APS Type 1), with support funding raised by the APS Type 1 Foundation

• Maureen A. Su MD, University of North Carolina at Chapel Hill

For the study of Homocystinuria due to Cystathionine Beta-Synthase Deficiency, with support funding raised from public donations

• Warren D. Kruger PhD, The Research Institute of Fox Chase Cancer Center

For the study of Malonic Aciduria, with support funding raised by the Hope Fund, Lundbeck “Raise Your Hand” campaign, and public donations

• Michael J. Wolfgang PhD, Johns Hopkins University School of Medicine

For the study of Stiff Person Syndrome, with support funding raised by the Lundbeck “Raise Your Hand” campaign and public donations

• Sarah Crisp PhD, University College, London

Grants are made possible by donations to NORD research funds and the assistance of rare disease medical experts who serve on NORD’s Medical Advisory Committee.

The National Organization for Rare Disorders (NORD) has awarded seven new research grants to fund rare disease research. These grants are in response to NORD’s 2016 Requests for Proposals. The 2017 Requests for Proposals will be published soon on the NORD website, where funding opportunities from NORD member organizations also are posted.

The new grant recipients are:

For the study of Alveolar Capillary Dysplasia with Misalignment of the Pulmonary Veins (ACD/MPV), with support funding raised by the David Ashwell Foundation and the Alveolar Capillary Dysplasia Association

• Przemyslaw Szafranski PhD, Baylor College of Medicine

For the study of Appendix Cancer and Pseudomyxoma Peritonei (PMP), with support funding raised by the Appendix Cancer Pseudomyxoma Peritonei Research Foundation

• Venkatesh Govindarajan PhD, Creighton University
• David L. Morris MD PhD, St. George Hospital, Australia

For the study of Autoimmune Polyglandular Syndrome Type 1 (APS Type 1), with support funding raised by the APS Type 1 Foundation

• Maureen A. Su MD, University of North Carolina at Chapel Hill

For the study of Homocystinuria due to Cystathionine Beta-Synthase Deficiency, with support funding raised from public donations

• Warren D. Kruger PhD, The Research Institute of Fox Chase Cancer Center

For the study of Malonic Aciduria, with support funding raised by the Hope Fund, Lundbeck “Raise Your Hand” campaign, and public donations

• Michael J. Wolfgang PhD, Johns Hopkins University School of Medicine

For the study of Stiff Person Syndrome, with support funding raised by the Lundbeck “Raise Your Hand” campaign and public donations

• Sarah Crisp PhD, University College, London

Grants are made possible by donations to NORD research funds and the assistance of rare disease medical experts who serve on NORD’s Medical Advisory Committee.

The National Organization for Rare Disorders (NORD) has awarded seven new research grants to fund rare disease research. These grants are in response to NORD’s 2016 Requests for Proposals. The 2017 Requests for Proposals will be published soon on the NORD website, where funding opportunities from NORD member organizations also are posted.

The new grant recipients are:

For the study of Alveolar Capillary Dysplasia with Misalignment of the Pulmonary Veins (ACD/MPV), with support funding raised by the David Ashwell Foundation and the Alveolar Capillary Dysplasia Association

• Przemyslaw Szafranski PhD, Baylor College of Medicine

For the study of Appendix Cancer and Pseudomyxoma Peritonei (PMP), with support funding raised by the Appendix Cancer Pseudomyxoma Peritonei Research Foundation

• Venkatesh Govindarajan PhD, Creighton University
• David L. Morris MD PhD, St. George Hospital, Australia

For the study of Autoimmune Polyglandular Syndrome Type 1 (APS Type 1), with support funding raised by the APS Type 1 Foundation

• Maureen A. Su MD, University of North Carolina at Chapel Hill

For the study of Homocystinuria due to Cystathionine Beta-Synthase Deficiency, with support funding raised from public donations

• Warren D. Kruger PhD, The Research Institute of Fox Chase Cancer Center

For the study of Malonic Aciduria, with support funding raised by the Hope Fund, Lundbeck “Raise Your Hand” campaign, and public donations

• Michael J. Wolfgang PhD, Johns Hopkins University School of Medicine

For the study of Stiff Person Syndrome, with support funding raised by the Lundbeck “Raise Your Hand” campaign and public donations

• Sarah Crisp PhD, University College, London

Grants are made possible by donations to NORD research funds and the assistance of rare disease medical experts who serve on NORD’s Medical Advisory Committee.

The Foundation for Prader-Willi Research has published a blog that discusses a new study which shows promising first steps toward genetic therapy for Prader-Willi syndrome.  

The Foundation for Prader-Willi Research has published a blog that discusses a new study which shows promising first steps toward genetic therapy for Prader-Willi syndrome.  

The Foundation for Prader-Willi Research has published a blog that discusses a new study which shows promising first steps toward genetic therapy for Prader-Willi syndrome.  

The second APS Type 1 Symposium for physicians and families will take place July 13 to15 at the State University of New York at Stony Brook. Additional information will be available from the APS Type 1 Foundation.

The second APS Type 1 Symposium for physicians and families will take place July 13 to15 at the State University of New York at Stony Brook. Additional information will be available from the APS Type 1 Foundation.

The second APS Type 1 Symposium for physicians and families will take place July 13 to15 at the State University of New York at Stony Brook. Additional information will be available from the APS Type 1 Foundation.

AAMDSIF has announced that its 2017 research grant portal is now open. Each year, the foundation’s medical advisors review research proposals from new and established investigators in the topic areas of aplastic anemia, myelodysplastic syndromes, and paroxysmal nocturnal hemoglobinuria. The deadline to submit an application is February 28, 2017.

AAMDSIF has announced that its 2017 research grant portal is now open. Each year, the foundation’s medical advisors review research proposals from new and established investigators in the topic areas of aplastic anemia, myelodysplastic syndromes, and paroxysmal nocturnal hemoglobinuria. The deadline to submit an application is February 28, 2017.

AAMDSIF has announced that its 2017 research grant portal is now open. Each year, the foundation’s medical advisors review research proposals from new and established investigators in the topic areas of aplastic anemia, myelodysplastic syndromes, and paroxysmal nocturnal hemoglobinuria. The deadline to submit an application is February 28, 2017.