News from NORD

Films from around the world featuring the challenges of life with a rare disease will be shown in the first-ever Rare Disease Film Festival to take place in Boston on Oct. 2-3. Tickets are on sale now and the event is open to all. Additional information is available here.

Films from around the world featuring the challenges of life with a rare disease will be shown in the first-ever Rare Disease Film Festival to take place in Boston on Oct. 2-3. Tickets are on sale now and the event is open to all. Additional information is available here.

Films from around the world featuring the challenges of life with a rare disease will be shown in the first-ever Rare Disease Film Festival to take place in Boston on Oct. 2-3. Tickets are on sale now and the event is open to all. Additional information is available here.

Do you currently treat patients with rare diseases? Do you suspect that you might have a patient with an undiagnosed rare disease? The National Organization for Rare Disorders (NORD) and the University of Massachusetts Medical School have designed two events for you and your colleagues to learn more about rare diseases and the available diagnostic tools.

An interactive half-day symposium on diagnostic hurdles, tools, and resources is planned for Sunday, October 16, 2017, at the Marriott Wardman Park Hotel in Washington DC. This event, “Finding a Zebra Among Many Horses,” will focus on challenges and resources related to rare disease diagnosis.

On the following two days, Oct. 16 and 17, NORD’s Rare Diseases and Orphan Products Breakthrough Summit will take place at the same location and is also CME-accredited. Topics to be covered will include:

• Assuring Patient Access: Future Outlook for Patient Assistance Programs
• The Challenge of Health Care Costs and Treatment Prices
• An Evergreen and Sustainable Approach to Orphan Drugs
• Next-Generation Treatments and Advancing Clinical Trials

The NORD Breakthrough Summit attracts approximately 600 participants from rare disease stakeholder groups, including clinicians, researchers, patient advocates, and government partners from the NIH and FDA.

Online registration is available now for either or both events.

Do you currently treat patients with rare diseases? Do you suspect that you might have a patient with an undiagnosed rare disease? The National Organization for Rare Disorders (NORD) and the University of Massachusetts Medical School have designed two events for you and your colleagues to learn more about rare diseases and the available diagnostic tools.

An interactive half-day symposium on diagnostic hurdles, tools, and resources is planned for Sunday, October 16, 2017, at the Marriott Wardman Park Hotel in Washington DC. This event, “Finding a Zebra Among Many Horses,” will focus on challenges and resources related to rare disease diagnosis.

On the following two days, Oct. 16 and 17, NORD’s Rare Diseases and Orphan Products Breakthrough Summit will take place at the same location and is also CME-accredited. Topics to be covered will include:

• Assuring Patient Access: Future Outlook for Patient Assistance Programs
• The Challenge of Health Care Costs and Treatment Prices
• An Evergreen and Sustainable Approach to Orphan Drugs
• Next-Generation Treatments and Advancing Clinical Trials

The NORD Breakthrough Summit attracts approximately 600 participants from rare disease stakeholder groups, including clinicians, researchers, patient advocates, and government partners from the NIH and FDA.

Online registration is available now for either or both events.

Do you currently treat patients with rare diseases? Do you suspect that you might have a patient with an undiagnosed rare disease? The National Organization for Rare Disorders (NORD) and the University of Massachusetts Medical School have designed two events for you and your colleagues to learn more about rare diseases and the available diagnostic tools.

An interactive half-day symposium on diagnostic hurdles, tools, and resources is planned for Sunday, October 16, 2017, at the Marriott Wardman Park Hotel in Washington DC. This event, “Finding a Zebra Among Many Horses,” will focus on challenges and resources related to rare disease diagnosis.

On the following two days, Oct. 16 and 17, NORD’s Rare Diseases and Orphan Products Breakthrough Summit will take place at the same location and is also CME-accredited. Topics to be covered will include:

• Assuring Patient Access: Future Outlook for Patient Assistance Programs
• The Challenge of Health Care Costs and Treatment Prices
• An Evergreen and Sustainable Approach to Orphan Drugs
• Next-Generation Treatments and Advancing Clinical Trials

The NORD Breakthrough Summit attracts approximately 600 participants from rare disease stakeholder groups, including clinicians, researchers, patient advocates, and government partners from the NIH and FDA.

Online registration is available now for either or both events.

Vestibular (inner ear and brain) disorders affect patients physically, mentally, and emotionally, causing chronic dizziness, vertigo, imbalance, tinnitus, depression, and anxiety. To promote awareness of these invisible conditions, the Vestibular Disorders Association celebrates Balance Awareness Week on September 18-24, 2017. Visit the website for more information.

Vestibular (inner ear and brain) disorders affect patients physically, mentally, and emotionally, causing chronic dizziness, vertigo, imbalance, tinnitus, depression, and anxiety. To promote awareness of these invisible conditions, the Vestibular Disorders Association celebrates Balance Awareness Week on September 18-24, 2017. Visit the website for more information.

Vestibular (inner ear and brain) disorders affect patients physically, mentally, and emotionally, causing chronic dizziness, vertigo, imbalance, tinnitus, depression, and anxiety. To promote awareness of these invisible conditions, the Vestibular Disorders Association celebrates Balance Awareness Week on September 18-24, 2017. Visit the website for more information.

A full-day multidisciplinary symposium on intracranial hypotension secondary to spinal cerebrospinal fluid (CSF) leak will bring together top clinicians and researchers to share the latest advances in diagnostics and treatments for this under-diagnosed but treatable secondary headache disorder. This event will take place Saturday, Oct. 14, 2017, in Santa Monica, California.

A full-day multidisciplinary symposium on intracranial hypotension secondary to spinal cerebrospinal fluid (CSF) leak will bring together top clinicians and researchers to share the latest advances in diagnostics and treatments for this under-diagnosed but treatable secondary headache disorder. This event will take place Saturday, Oct. 14, 2017, in Santa Monica, California.

A full-day multidisciplinary symposium on intracranial hypotension secondary to spinal cerebrospinal fluid (CSF) leak will bring together top clinicians and researchers to share the latest advances in diagnostics and treatments for this under-diagnosed but treatable secondary headache disorder. This event will take place Saturday, Oct. 14, 2017, in Santa Monica, California.

The Neuroendocrine Tumor Research Foundation (NETRF) has issued an invitation for Letters of Intent for 2017 research grants. Proposals are being sought with the potential to transform understanding of neuroendocrine tumors and/or lead to improved treatments and diagnostics for patients. Letters of Intent may focus on any type of neuroendocrine tumor and may be for basic, translational, or clinical research.

The Neuroendocrine Tumor Research Foundation (NETRF) has issued an invitation for Letters of Intent for 2017 research grants. Proposals are being sought with the potential to transform understanding of neuroendocrine tumors and/or lead to improved treatments and diagnostics for patients. Letters of Intent may focus on any type of neuroendocrine tumor and may be for basic, translational, or clinical research.

The Neuroendocrine Tumor Research Foundation (NETRF) has issued an invitation for Letters of Intent for 2017 research grants. Proposals are being sought with the potential to transform understanding of neuroendocrine tumors and/or lead to improved treatments and diagnostics for patients. Letters of Intent may focus on any type of neuroendocrine tumor and may be for basic, translational, or clinical research.

The Multiple System Atrophy (MSA) Coalition Patient/Family Conference will take place October 13-14, 2017, in Nashville.

The Multiple System Atrophy (MSA) Coalition Patient/Family Conference will take place October 13-14, 2017, in Nashville.

The Multiple System Atrophy (MSA) Coalition Patient/Family Conference will take place October 13-14, 2017, in Nashville.

The Hyper IgM Foundation recently published a blog promoting better understanding of Hyper IgM syndrome, a rare, genetic, immunodeficiency disorder. In particular, they are seeking to provide information for patients and families going through stem cell transplantation.

The Hyper IgM Foundation recently published a blog promoting better understanding of Hyper IgM syndrome, a rare, genetic, immunodeficiency disorder. In particular, they are seeking to provide information for patients and families going through stem cell transplantation.

The Hyper IgM Foundation recently published a blog promoting better understanding of Hyper IgM syndrome, a rare, genetic, immunodeficiency disorder. In particular, they are seeking to provide information for patients and families going through stem cell transplantation.

The cureCADASIL Association has announced the publication of a study by Dr. Arboleda-Velasquez in the Journal of Experimental Medicine. CADASIL is a disorder of the small blood vessels that causes subcortical transient ischemic attacks or strokes and progresses to early vascular dementia.

The cureCADASIL Association has announced the publication of a study by Dr. Arboleda-Velasquez in the Journal of Experimental Medicine. CADASIL is a disorder of the small blood vessels that causes subcortical transient ischemic attacks or strokes and progresses to early vascular dementia.

The cureCADASIL Association has announced the publication of a study by Dr. Arboleda-Velasquez in the Journal of Experimental Medicine. CADASIL is a disorder of the small blood vessels that causes subcortical transient ischemic attacks or strokes and progresses to early vascular dementia.

The Alpha-1 Foundation has announced its funding opportunities in its 2017-2018 grants cycle. In-cycle Letters of Intent are due by September 22, 2017, and may be submitted online.

The Alpha-1 Foundation has announced its funding opportunities in its 2017-2018 grants cycle. In-cycle Letters of Intent are due by September 22, 2017, and may be submitted online.

The Alpha-1 Foundation has announced its funding opportunities in its 2017-2018 grants cycle. In-cycle Letters of Intent are due by September 22, 2017, and may be submitted online.

All Things Kabuki, an organization providing awareness, education, and support on behalf of those affected by Kabuki syndrome, is NORD’s newest member organization. Read NORD’s Rare Disease Database report on Kabuki syndrome.

All Things Kabuki, an organization providing awareness, education, and support on behalf of those affected by Kabuki syndrome, is NORD’s newest member organization. Read NORD’s Rare Disease Database report on Kabuki syndrome.

All Things Kabuki, an organization providing awareness, education, and support on behalf of those affected by Kabuki syndrome, is NORD’s newest member organization. Read NORD’s Rare Disease Database report on Kabuki syndrome.