News from NORD

The NIH Undiagnosed Diseases Network (UDN) will launch a webinar series on March 8, 2018  (1–2 p.m. ET) that will feature descriptions of clinical phenotype and diagnostic evaluations of UDN patients. Webinar participants will be able to ask questions and offer insights on the presented cases. Registration is free but required using this link.  This activity has been approved for free AMA PRA Category 1 Credit.  Contact [email protected] with questions.

The NIH Undiagnosed Diseases Network (UDN) will launch a webinar series on March 8, 2018  (1–2 p.m. ET) that will feature descriptions of clinical phenotype and diagnostic evaluations of UDN patients. Webinar participants will be able to ask questions and offer insights on the presented cases. Registration is free but required using this link.  This activity has been approved for free AMA PRA Category 1 Credit.  Contact [email protected] with questions.

The NIH Undiagnosed Diseases Network (UDN) will launch a webinar series on March 8, 2018  (1–2 p.m. ET) that will feature descriptions of clinical phenotype and diagnostic evaluations of UDN patients. Webinar participants will be able to ask questions and offer insights on the presented cases. Registration is free but required using this link.  This activity has been approved for free AMA PRA Category 1 Credit.  Contact [email protected] with questions.

The Food and Drug Administration (FDA) has released a draft guidance describing a possible new approach for companies to collaborate and test multiple drug products in the same clinical trials. Public comment is welcomed.

FDA also has issued a guidance on clarification of orphan designation of drugs and biologics for pediatric subpopulations of common diseases.

The Food and Drug Administration (FDA) has released a draft guidance describing a possible new approach for companies to collaborate and test multiple drug products in the same clinical trials. Public comment is welcomed.

FDA also has issued a guidance on clarification of orphan designation of drugs and biologics for pediatric subpopulations of common diseases.

The Food and Drug Administration (FDA) has released a draft guidance describing a possible new approach for companies to collaborate and test multiple drug products in the same clinical trials. Public comment is welcomed.

FDA also has issued a guidance on clarification of orphan designation of drugs and biologics for pediatric subpopulations of common diseases.

The Tax Cuts and Jobs Act passed by Congress and signed into law by President Trump includes a reduction of the Orphan Drug Tax Credit (ODTC), a repeal of the Affordable Care Act’s individual mandate, and a temporary bolstering of the Medical Expense Deduction. While NORD supports the temporary strengthening of the Medical Expense Deduction, it opposed the repeal of the individual mandate and the reduction of the ODTC. Thanks to the work of rare disease advocates joining NORD in support for the ODTC, the tax credit was not repealed entirely, as was initially suggested, but rather was cut in half. NORD is grateful for the support it received on this issue and will continue to work to preserve the important orphan drug incentives in 2018.

The Tax Cuts and Jobs Act passed by Congress and signed into law by President Trump includes a reduction of the Orphan Drug Tax Credit (ODTC), a repeal of the Affordable Care Act’s individual mandate, and a temporary bolstering of the Medical Expense Deduction. While NORD supports the temporary strengthening of the Medical Expense Deduction, it opposed the repeal of the individual mandate and the reduction of the ODTC. Thanks to the work of rare disease advocates joining NORD in support for the ODTC, the tax credit was not repealed entirely, as was initially suggested, but rather was cut in half. NORD is grateful for the support it received on this issue and will continue to work to preserve the important orphan drug incentives in 2018.

The Tax Cuts and Jobs Act passed by Congress and signed into law by President Trump includes a reduction of the Orphan Drug Tax Credit (ODTC), a repeal of the Affordable Care Act’s individual mandate, and a temporary bolstering of the Medical Expense Deduction. While NORD supports the temporary strengthening of the Medical Expense Deduction, it opposed the repeal of the individual mandate and the reduction of the ODTC. Thanks to the work of rare disease advocates joining NORD in support for the ODTC, the tax credit was not repealed entirely, as was initially suggested, but rather was cut in half. NORD is grateful for the support it received on this issue and will continue to work to preserve the important orphan drug incentives in 2018.

2018 marks the 35th anniversary of the Orphan Drug Act (ODA) and of NORD. The ODA was signed into law by President Ronald Reagan on January 4, 1983.  Exactly four months later, NORD was formally established by the patient organization leaders who had provided advocacy for the ODA. The ODA provides critically important financial incentives to encourage development of treatments for rare diseases. Major events during the anniversary year will include NORD’s Rare Impact Awards in May and Rare Summit in October. Visit the NORD website often this year for information about those and other anniversary activities.

2018 marks the 35th anniversary of the Orphan Drug Act (ODA) and of NORD. The ODA was signed into law by President Ronald Reagan on January 4, 1983.  Exactly four months later, NORD was formally established by the patient organization leaders who had provided advocacy for the ODA. The ODA provides critically important financial incentives to encourage development of treatments for rare diseases. Major events during the anniversary year will include NORD’s Rare Impact Awards in May and Rare Summit in October. Visit the NORD website often this year for information about those and other anniversary activities.

2018 marks the 35th anniversary of the Orphan Drug Act (ODA) and of NORD. The ODA was signed into law by President Ronald Reagan on January 4, 1983.  Exactly four months later, NORD was formally established by the patient organization leaders who had provided advocacy for the ODA. The ODA provides critically important financial incentives to encourage development of treatments for rare diseases. Major events during the anniversary year will include NORD’s Rare Impact Awards in May and Rare Summit in October. Visit the NORD website often this year for information about those and other anniversary activities.

NORD research grant opportunities are posted throughout the year as funds become available for research on specific rare diseases. Researchers should visit the website periodically to learn whether any new requests for proposals (RFPs) have been posted. Information about the NORD Research Program and current RFPs may be found here.

NORD research grant opportunities are posted throughout the year as funds become available for research on specific rare diseases. Researchers should visit the website periodically to learn whether any new requests for proposals (RFPs) have been posted. Information about the NORD Research Program and current RFPs may be found here.

NORD research grant opportunities are posted throughout the year as funds become available for research on specific rare diseases. Researchers should visit the website periodically to learn whether any new requests for proposals (RFPs) have been posted. Information about the NORD Research Program and current RFPs may be found here.

NORD has awarded research grants to the following scientists and institutions:

• Arun Pradhan, PhD, Cincinnati Children’s Hospital Medical Center, Cincinnati, Ohio
• J. Silvio Gutkind, PhD, University of California, San Diego, California
• D. Scott Merrell, PhD, Uniformed Services University of the Health Sciences, Bethesda, Maryland
• Marc Pocard, MD, PhD, Institut national de la santé et de la recherche médicale (Inserm), Paris
• Traci L. Testerman, PhD, University of South Carolina School of Medicine, Columbia, South Carolina

These grants are for studies of alveolar capillary dysplasia with misalignment of the pulmonary veins (with support from the David Ashwell Foundation, Alveolar Capillary Dysplasia Association and William Akers Jr. and Georgia O. Akers Private Foundation) and appendix cancer pseudomyxoma peritonei (with support from the Appendix Cancer Pseudomyxoma Peritonei Research Foundation).

NORD has awarded research grants to the following scientists and institutions:

• Arun Pradhan, PhD, Cincinnati Children’s Hospital Medical Center, Cincinnati, Ohio
• J. Silvio Gutkind, PhD, University of California, San Diego, California
• D. Scott Merrell, PhD, Uniformed Services University of the Health Sciences, Bethesda, Maryland
• Marc Pocard, MD, PhD, Institut national de la santé et de la recherche médicale (Inserm), Paris
• Traci L. Testerman, PhD, University of South Carolina School of Medicine, Columbia, South Carolina

These grants are for studies of alveolar capillary dysplasia with misalignment of the pulmonary veins (with support from the David Ashwell Foundation, Alveolar Capillary Dysplasia Association and William Akers Jr. and Georgia O. Akers Private Foundation) and appendix cancer pseudomyxoma peritonei (with support from the Appendix Cancer Pseudomyxoma Peritonei Research Foundation).

NORD has awarded research grants to the following scientists and institutions:

• Arun Pradhan, PhD, Cincinnati Children’s Hospital Medical Center, Cincinnati, Ohio
• J. Silvio Gutkind, PhD, University of California, San Diego, California
• D. Scott Merrell, PhD, Uniformed Services University of the Health Sciences, Bethesda, Maryland
• Marc Pocard, MD, PhD, Institut national de la santé et de la recherche médicale (Inserm), Paris
• Traci L. Testerman, PhD, University of South Carolina School of Medicine, Columbia, South Carolina

These grants are for studies of alveolar capillary dysplasia with misalignment of the pulmonary veins (with support from the David Ashwell Foundation, Alveolar Capillary Dysplasia Association and William Akers Jr. and Georgia O. Akers Private Foundation) and appendix cancer pseudomyxoma peritonei (with support from the Appendix Cancer Pseudomyxoma Peritonei Research Foundation).

Nominate a colleague, patient, caregiver, or organization for a NORD Rare Impact Award online by January 12, 2018. These awards honor individuals or organizations for improving the lives of those affected by rare diseases. The awards are presented at NORD’s Rare Impact Awards event in Washington, DC in May. Over the years, those honored have included members of Congress, senior officials from FDA and NIH, clinicians, researchers, medical societies, patient organizations, and others whose work has had a beneficial impact on the community. More info.

Nominate a colleague, patient, caregiver, or organization for a NORD Rare Impact Award online by January 12, 2018. These awards honor individuals or organizations for improving the lives of those affected by rare diseases. The awards are presented at NORD’s Rare Impact Awards event in Washington, DC in May. Over the years, those honored have included members of Congress, senior officials from FDA and NIH, clinicians, researchers, medical societies, patient organizations, and others whose work has had a beneficial impact on the community. More info.

Nominate a colleague, patient, caregiver, or organization for a NORD Rare Impact Award online by January 12, 2018. These awards honor individuals or organizations for improving the lives of those affected by rare diseases. The awards are presented at NORD’s Rare Impact Awards event in Washington, DC in May. Over the years, those honored have included members of Congress, senior officials from FDA and NIH, clinicians, researchers, medical societies, patient organizations, and others whose work has had a beneficial impact on the community. More info.

In anticipation of “Rare Disease Day” on February 28, 2018, NORD will be hosting a “7,000 Miles Campaign” for everyone interested in showing support for rare disease patients. The campaign will run from February 1 through February 28, and the goal will be for participants to walk, run, or cycle 7,000 miles during that time period. Funds raised will support NORD’s programs of advocacy, education, research, and patient services.  Everyone seeking to improve the lives of rare disease patients is invited to participate and to share information about the campaign with others. More info.

In anticipation of “Rare Disease Day” on February 28, 2018, NORD will be hosting a “7,000 Miles Campaign” for everyone interested in showing support for rare disease patients. The campaign will run from February 1 through February 28, and the goal will be for participants to walk, run, or cycle 7,000 miles during that time period. Funds raised will support NORD’s programs of advocacy, education, research, and patient services.  Everyone seeking to improve the lives of rare disease patients is invited to participate and to share information about the campaign with others. More info.

In anticipation of “Rare Disease Day” on February 28, 2018, NORD will be hosting a “7,000 Miles Campaign” for everyone interested in showing support for rare disease patients. The campaign will run from February 1 through February 28, and the goal will be for participants to walk, run, or cycle 7,000 miles during that time period. Funds raised will support NORD’s programs of advocacy, education, research, and patient services.  Everyone seeking to improve the lives of rare disease patients is invited to participate and to share information about the campaign with others. More info.

The Osteogenesis Imperfecta Foundation (OIF) will host its biennial national conference in Baltimore July 13-15, 2018. The program will feature sessions on medical advances and practical living guidance, along with forums with leading experts in osteogenesis imperfecta care and research. More info.  

The Osteogenesis Imperfecta Foundation (OIF) will host its biennial national conference in Baltimore July 13-15, 2018. The program will feature sessions on medical advances and practical living guidance, along with forums with leading experts in osteogenesis imperfecta care and research. More info.  

The Osteogenesis Imperfecta Foundation (OIF) will host its biennial national conference in Baltimore July 13-15, 2018. The program will feature sessions on medical advances and practical living guidance, along with forums with leading experts in osteogenesis imperfecta care and research. More info.  

The National Tay-Sachs & Allied Diseases Association (NTSAD), in partnership with the Cure Tay-Sachs Foundation, is currently accepting pre-applications for research grants, with a deadline of Jan. 12, 2018. NTSAD funds research that may lead to treatments for lysosomal storage diseases or leukodystrophies impacting the central nervous system. More info.

The National Tay-Sachs & Allied Diseases Association (NTSAD), in partnership with the Cure Tay-Sachs Foundation, is currently accepting pre-applications for research grants, with a deadline of Jan. 12, 2018. NTSAD funds research that may lead to treatments for lysosomal storage diseases or leukodystrophies impacting the central nervous system. More info.

The National Tay-Sachs & Allied Diseases Association (NTSAD), in partnership with the Cure Tay-Sachs Foundation, is currently accepting pre-applications for research grants, with a deadline of Jan. 12, 2018. NTSAD funds research that may lead to treatments for lysosomal storage diseases or leukodystrophies impacting the central nervous system. More info.