News from NORD

At its recent Breakthrough Summit, NORD released the first-ever State Progress Report, which documents progress in all 50 states regarding implementing policies pertinent to rare diseases. This report will serve as a baseline to map progress toward future improvement in the states. It is the first comprehensive resource to document inconsistencies among states in four key areas: medical foods coverage, prescription drug cost-sharing requirements, newborn screening, and Medicaid eligibility levels.

The 65-page document contains an executive and national summary, key policy sections, detailed appendices with state-by-state breakdowns, maps, contacts, and resources. The report is also a toolkit providing resources for advocacy on the key issues.

At its recent Breakthrough Summit, NORD released the first-ever State Progress Report, which documents progress in all 50 states regarding implementing policies pertinent to rare diseases. This report will serve as a baseline to map progress toward future improvement in the states. It is the first comprehensive resource to document inconsistencies among states in four key areas: medical foods coverage, prescription drug cost-sharing requirements, newborn screening, and Medicaid eligibility levels.

The 65-page document contains an executive and national summary, key policy sections, detailed appendices with state-by-state breakdowns, maps, contacts, and resources. The report is also a toolkit providing resources for advocacy on the key issues.

At its recent Breakthrough Summit, NORD released the first-ever State Progress Report, which documents progress in all 50 states regarding implementing policies pertinent to rare diseases. This report will serve as a baseline to map progress toward future improvement in the states. It is the first comprehensive resource to document inconsistencies among states in four key areas: medical foods coverage, prescription drug cost-sharing requirements, newborn screening, and Medicaid eligibility levels.

The 65-page document contains an executive and national summary, key policy sections, detailed appendices with state-by-state breakdowns, maps, contacts, and resources. The report is also a toolkit providing resources for advocacy on the key issues.

The Consortium of Multiple Sclerosis Centers (CMSC), a professional and educational organization for clinicians and researchers treating MS patients or studying MS, has issued a position statement asserting that prescribers must retain the right to decide on the best treatment and medication for each individual MS patient. The Consortium membership includes more than 7,000 international clinicians and researchers committed to MS care, more than 60 Veteran’s Administration MS programs, and 225 MS centers in the US, Canada, and Europe.

The Consortium of Multiple Sclerosis Centers (CMSC), a professional and educational organization for clinicians and researchers treating MS patients or studying MS, has issued a position statement asserting that prescribers must retain the right to decide on the best treatment and medication for each individual MS patient. The Consortium membership includes more than 7,000 international clinicians and researchers committed to MS care, more than 60 Veteran’s Administration MS programs, and 225 MS centers in the US, Canada, and Europe.

The Consortium of Multiple Sclerosis Centers (CMSC), a professional and educational organization for clinicians and researchers treating MS patients or studying MS, has issued a position statement asserting that prescribers must retain the right to decide on the best treatment and medication for each individual MS patient. The Consortium membership includes more than 7,000 international clinicians and researchers committed to MS care, more than 60 Veteran’s Administration MS programs, and 225 MS centers in the US, Canada, and Europe.

Learning sessions have been scheduled for Nov. 18 to discuss how volunteers are protected in research and possible changes to the federal regulations. Individuals may participate in person or via live webinar.

Learning sessions have been scheduled for Nov. 18 to discuss how volunteers are protected in research and possible changes to the federal regulations. Individuals may participate in person or via live webinar.

Learning sessions have been scheduled for Nov. 18 to discuss how volunteers are protected in research and possible changes to the federal regulations. Individuals may participate in person or via live webinar.

The Office of Orphan Products Development (OOPD) at the Food and Drug Administration (FDA) has posted information for those interested in applying for orphan product development grants. These are grants to support the clinical development of products for use in rare diseases or conditions for which no current therapy exists or when the product being developed will be superior to existing treatments.

The Office of Orphan Products Development (OOPD) at the Food and Drug Administration (FDA) has posted information for those interested in applying for orphan product development grants. These are grants to support the clinical development of products for use in rare diseases or conditions for which no current therapy exists or when the product being developed will be superior to existing treatments.

The Office of Orphan Products Development (OOPD) at the Food and Drug Administration (FDA) has posted information for those interested in applying for orphan product development grants. These are grants to support the clinical development of products for use in rare diseases or conditions for which no current therapy exists or when the product being developed will be superior to existing treatments.

The National Center for Advancing Translational Sciences is now accepting proposals on a rolling basis to collaborate with Bridging Interventional Development Gaps and Therapeutics for Rare and Neglected Diseases program scientists.

The National Center for Advancing Translational Sciences is now accepting proposals on a rolling basis to collaborate with Bridging Interventional Development Gaps and Therapeutics for Rare and Neglected Diseases program scientists.

The National Center for Advancing Translational Sciences is now accepting proposals on a rolling basis to collaborate with Bridging Interventional Development Gaps and Therapeutics for Rare and Neglected Diseases program scientists.

The National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health (NIH) is collecting stakeholder input on the scientific and operational opportunities, challenges, and research needs in translational science. The information will help determine the Center’s strategic priorities and inform the development of a five-year strategic plan. The deadline to submit input is January 8, 2016.

The National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health (NIH) is collecting stakeholder input on the scientific and operational opportunities, challenges, and research needs in translational science. The information will help determine the Center’s strategic priorities and inform the development of a five-year strategic plan. The deadline to submit input is January 8, 2016.

The National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health (NIH) is collecting stakeholder input on the scientific and operational opportunities, challenges, and research needs in translational science. The information will help determine the Center’s strategic priorities and inform the development of a five-year strategic plan. The deadline to submit input is January 8, 2016.

At its recent Breakthrough Summit, NORD released the first-ever State Progress Report, which documents progress in all 50 states regarding implementing policies pertinent to rare diseases. This report will serve as a baseline to map progress toward future improvement in the states. It is the first comprehensive resource to document inconsistencies among states in four key areas: medical foods coverage, prescription drug cost-sharing requirements, newborn screening, and Medicaid eligibility levels.

The 65-page document contains an executive and national summary, key policy sections, detailed appendices with state-by-state breakdowns, maps, contacts, and resources. The report is also a toolkit providing resources for advocacy on the key issues.

At its recent Breakthrough Summit, NORD released the first-ever State Progress Report, which documents progress in all 50 states regarding implementing policies pertinent to rare diseases. This report will serve as a baseline to map progress toward future improvement in the states. It is the first comprehensive resource to document inconsistencies among states in four key areas: medical foods coverage, prescription drug cost-sharing requirements, newborn screening, and Medicaid eligibility levels.

The 65-page document contains an executive and national summary, key policy sections, detailed appendices with state-by-state breakdowns, maps, contacts, and resources. The report is also a toolkit providing resources for advocacy on the key issues.

At its recent Breakthrough Summit, NORD released the first-ever State Progress Report, which documents progress in all 50 states regarding implementing policies pertinent to rare diseases. This report will serve as a baseline to map progress toward future improvement in the states. It is the first comprehensive resource to document inconsistencies among states in four key areas: medical foods coverage, prescription drug cost-sharing requirements, newborn screening, and Medicaid eligibility levels.

The 65-page document contains an executive and national summary, key policy sections, detailed appendices with state-by-state breakdowns, maps, contacts, and resources. The report is also a toolkit providing resources for advocacy on the key issues.

The Undiagnosed Diseases Network (UDN) Gateway is an online patient application portal for all clinical sites in the UDN, including the original Undiagnosed Diseases Program at the NIH Clinical Center in Bethesda. The core mission of the UDN is to diagnose patients who suffer from undiagnosed conditions despite extensive clinical investigation. More information.

The Undiagnosed Diseases Network (UDN) Gateway is an online patient application portal for all clinical sites in the UDN, including the original Undiagnosed Diseases Program at the NIH Clinical Center in Bethesda. The core mission of the UDN is to diagnose patients who suffer from undiagnosed conditions despite extensive clinical investigation. More information.

The Undiagnosed Diseases Network (UDN) Gateway is an online patient application portal for all clinical sites in the UDN, including the original Undiagnosed Diseases Program at the NIH Clinical Center in Bethesda. The core mission of the UDN is to diagnose patients who suffer from undiagnosed conditions despite extensive clinical investigation. More information.

The Food and Drug Administration has released a draft guidance document for navigating the unique challenges of developing treatments for rare diseases. It emphasizes the importance of understanding the natural history of a disease, creating study designs with clinically meaningful end points, establishing product safety and effectiveness, and ensuring product quality.

The Food and Drug Administration has released a draft guidance document for navigating the unique challenges of developing treatments for rare diseases. It emphasizes the importance of understanding the natural history of a disease, creating study designs with clinically meaningful end points, establishing product safety and effectiveness, and ensuring product quality.

The Food and Drug Administration has released a draft guidance document for navigating the unique challenges of developing treatments for rare diseases. It emphasizes the importance of understanding the natural history of a disease, creating study designs with clinically meaningful end points, establishing product safety and effectiveness, and ensuring product quality.

NORD supports this bipartisan bill, introduced in the US Senate, to advance the development of targeted drugs for patients with serious or life-threatening rare genetic diseases. The bill is intended to maintain FDA’s strong safety and effectiveness standards, while clarifying the agency’s authority to consider research supporting previously approved targeted treatments. More information.

NORD supports this bipartisan bill, introduced in the US Senate, to advance the development of targeted drugs for patients with serious or life-threatening rare genetic diseases. The bill is intended to maintain FDA’s strong safety and effectiveness standards, while clarifying the agency’s authority to consider research supporting previously approved targeted treatments. More information.

NORD supports this bipartisan bill, introduced in the US Senate, to advance the development of targeted drugs for patients with serious or life-threatening rare genetic diseases. The bill is intended to maintain FDA’s strong safety and effectiveness standards, while clarifying the agency’s authority to consider research supporting previously approved targeted treatments. More information.