News from NORD

The National Organization for Rare Disorders (NORD) and several other leading patient advocacy organizations have issued a joint statement opposing the American Health Care Act (AHCA). The patient advocates say the AHCA would:

• profoundly reduce health care coverage for millions of Americans
• weaken key consumer protections
• enable insurers to charge higher prices to those with pre-existing conditions and
• increase out-of-pocket costs for the sickest and oldest individuals

The organizations that joined together to issue the statement in addition NORD are: American Cancer Society Cancer Action Network, American Diabetes Association, American Heart Association, American Lung Association, Cystic Fibrosis Foundation, Juvenile Diabetes Research Foundation, March of Dimes, National MS Society, and WomenHeart: The National Coalition for Women with Heart Disease.

“As Congress considers this legislation,” the statement says, “we challenge lawmakers to remember their commitment to their constituents and the American people to protect lifesaving health care for millions of Americans, including those who struggle every day with chronic and other major health conditions. We stand ready to work with Congress toward a proposal that ensures all Americans have affordable access to the care they need.” Read the entire statement.

The National Organization for Rare Disorders (NORD) and several other leading patient advocacy organizations have issued a joint statement opposing the American Health Care Act (AHCA). The patient advocates say the AHCA would:

• profoundly reduce health care coverage for millions of Americans
• weaken key consumer protections
• enable insurers to charge higher prices to those with pre-existing conditions and
• increase out-of-pocket costs for the sickest and oldest individuals

The organizations that joined together to issue the statement in addition NORD are: American Cancer Society Cancer Action Network, American Diabetes Association, American Heart Association, American Lung Association, Cystic Fibrosis Foundation, Juvenile Diabetes Research Foundation, March of Dimes, National MS Society, and WomenHeart: The National Coalition for Women with Heart Disease.

“As Congress considers this legislation,” the statement says, “we challenge lawmakers to remember their commitment to their constituents and the American people to protect lifesaving health care for millions of Americans, including those who struggle every day with chronic and other major health conditions. We stand ready to work with Congress toward a proposal that ensures all Americans have affordable access to the care they need.” Read the entire statement.

The National Organization for Rare Disorders (NORD) and several other leading patient advocacy organizations have issued a joint statement opposing the American Health Care Act (AHCA). The patient advocates say the AHCA would:

• profoundly reduce health care coverage for millions of Americans
• weaken key consumer protections
• enable insurers to charge higher prices to those with pre-existing conditions and
• increase out-of-pocket costs for the sickest and oldest individuals

The organizations that joined together to issue the statement in addition NORD are: American Cancer Society Cancer Action Network, American Diabetes Association, American Heart Association, American Lung Association, Cystic Fibrosis Foundation, Juvenile Diabetes Research Foundation, March of Dimes, National MS Society, and WomenHeart: The National Coalition for Women with Heart Disease.

“As Congress considers this legislation,” the statement says, “we challenge lawmakers to remember their commitment to their constituents and the American people to protect lifesaving health care for millions of Americans, including those who struggle every day with chronic and other major health conditions. We stand ready to work with Congress toward a proposal that ensures all Americans have affordable access to the care they need.” Read the entire statement.

The Foundation for Prader-Willi Research has published a blog and brochure regarding recent findings related to use of growth hormone for those affected by Prader-Willi syndrome.

The Foundation for Prader-Willi Research has published a blog and brochure regarding recent findings related to use of growth hormone for those affected by Prader-Willi syndrome.

The Foundation for Prader-Willi Research has published a blog and brochure regarding recent findings related to use of growth hormone for those affected by Prader-Willi syndrome.

NORD Chief Operating Officer Pamela Gavin will speak at a symposium to be hosted by the University of Rochester on May 12 on Technology and Rare Neurological Diseases. Registration is open for this event, which will focus on novel applications of technology to accelerate the development of new therapeutics for individuals with rare neurological diseases.

NORD Chief Operating Officer Pamela Gavin will speak at a symposium to be hosted by the University of Rochester on May 12 on Technology and Rare Neurological Diseases. Registration is open for this event, which will focus on novel applications of technology to accelerate the development of new therapeutics for individuals with rare neurological diseases.

NORD Chief Operating Officer Pamela Gavin will speak at a symposium to be hosted by the University of Rochester on May 12 on Technology and Rare Neurological Diseases. Registration is open for this event, which will focus on novel applications of technology to accelerate the development of new therapeutics for individuals with rare neurological diseases.

A natural history registry that will provide a complete picture of each patient’s experience with pemphigus and pemphigoid has been launched by the International Pemphigus and Pemphigoid Foundation with NORD. This study will be open to patient worldwide to advance understanding and treatments for these rare conditions. The registry will be maintained on a platform developed by NORD in line with its mission to seek treatments or cures for all rare diseases. The project is supported by a cooperative agreement between NORD and the US Food and Drug Administration (FDA).

A natural history registry that will provide a complete picture of each patient’s experience with pemphigus and pemphigoid has been launched by the International Pemphigus and Pemphigoid Foundation with NORD. This study will be open to patient worldwide to advance understanding and treatments for these rare conditions. The registry will be maintained on a platform developed by NORD in line with its mission to seek treatments or cures for all rare diseases. The project is supported by a cooperative agreement between NORD and the US Food and Drug Administration (FDA).

A natural history registry that will provide a complete picture of each patient’s experience with pemphigus and pemphigoid has been launched by the International Pemphigus and Pemphigoid Foundation with NORD. This study will be open to patient worldwide to advance understanding and treatments for these rare conditions. The registry will be maintained on a platform developed by NORD in line with its mission to seek treatments or cures for all rare diseases. The project is supported by a cooperative agreement between NORD and the US Food and Drug Administration (FDA).

NORD is once again partnering with The Hole in the Wall Gang Camp on a rare disease summer family camp in Connecticut. Families living in the Northeast may submit applications now for this free weekend of fun. The camp is located in Ashford, CT, and will take place June 1 to 4.

NORD is once again partnering with The Hole in the Wall Gang Camp on a rare disease summer family camp in Connecticut. Families living in the Northeast may submit applications now for this free weekend of fun. The camp is located in Ashford, CT, and will take place June 1 to 4.

NORD is once again partnering with The Hole in the Wall Gang Camp on a rare disease summer family camp in Connecticut. Families living in the Northeast may submit applications now for this free weekend of fun. The camp is located in Ashford, CT, and will take place June 1 to 4.

A rare disease film festival will take place October 3 in Boston. Filmmakers whose work addresses issues of concern to rare disease patients may submit their work. By shining a light on the challenges that people with rare diseases and their loved ones face, the organizers hope to inspire people to take positive action to help others.

A rare disease film festival will take place October 3 in Boston. Filmmakers whose work addresses issues of concern to rare disease patients may submit their work. By shining a light on the challenges that people with rare diseases and their loved ones face, the organizers hope to inspire people to take positive action to help others.

A rare disease film festival will take place October 3 in Boston. Filmmakers whose work addresses issues of concern to rare disease patients may submit their work. By shining a light on the challenges that people with rare diseases and their loved ones face, the organizers hope to inspire people to take positive action to help others.

NORD’s annual Rare Diseases and Orphan Products Breakthrough Summit will take place October 16-17 in Washington DC. The event brings together more than 500 attendees, including medical professionals, patient advocates, NIH and FDA officials, and those developing diagnostics and treatments for patients with rare diseases. The Summit is open to all. Sign up to receive updates on the Summit and/or other upcoming events.

NORD’s annual Rare Diseases and Orphan Products Breakthrough Summit will take place October 16-17 in Washington DC. The event brings together more than 500 attendees, including medical professionals, patient advocates, NIH and FDA officials, and those developing diagnostics and treatments for patients with rare diseases. The Summit is open to all. Sign up to receive updates on the Summit and/or other upcoming events.

NORD’s annual Rare Diseases and Orphan Products Breakthrough Summit will take place October 16-17 in Washington DC. The event brings together more than 500 attendees, including medical professionals, patient advocates, NIH and FDA officials, and those developing diagnostics and treatments for patients with rare diseases. The Summit is open to all. Sign up to receive updates on the Summit and/or other upcoming events.

NORD and Neurology Reviews together have published the third annual Rare Neurological Disease Special Report in print and digital editions to promote awareness and understanding of rare neurological diseases among medical professionals. The publication includes articles on the efficacy of cannabis in treating children with rare and refractory epilepsy, delays in diagnosis and treatment of infantile spasms, telemedicine, new and potential therapies for rare neuromuscular disorders, and the clinical therapeutic potential of gene therapy.

NORD and Neurology Reviews together have published the third annual Rare Neurological Disease Special Report in print and digital editions to promote awareness and understanding of rare neurological diseases among medical professionals. The publication includes articles on the efficacy of cannabis in treating children with rare and refractory epilepsy, delays in diagnosis and treatment of infantile spasms, telemedicine, new and potential therapies for rare neuromuscular disorders, and the clinical therapeutic potential of gene therapy.

NORD and Neurology Reviews together have published the third annual Rare Neurological Disease Special Report in print and digital editions to promote awareness and understanding of rare neurological diseases among medical professionals. The publication includes articles on the efficacy of cannabis in treating children with rare and refractory epilepsy, delays in diagnosis and treatment of infantile spasms, telemedicine, new and potential therapies for rare neuromuscular disorders, and the clinical therapeutic potential of gene therapy.

Honorees at NORD’s 2017 Rare Impact Awards will include three clinicians and researchers with expertise in rare diseases. They are Robert Desnick, MD, PhD, of Mount Sinai Hospital and the Icahn School of Medicine in New York City; Frederick Kaplan, MD, of the Perelman School of Medicine at the University of Pennsylvania; and Cynthia Tifft, MD, PhD, of the National Institutes of Health. The three, along with other honorees, will be recognized for their work to improve the lives of patients affected by rare diseases.

The annual charity event will take place this year on May 18 at the Ronald Reagan Building and International Trade Center Amphitheatre in Washington DC. The event is open to the public and is a fundraiser to support NORD programs and services for all individuals who have rare diseases.

Honorees at NORD’s 2017 Rare Impact Awards will include three clinicians and researchers with expertise in rare diseases. They are Robert Desnick, MD, PhD, of Mount Sinai Hospital and the Icahn School of Medicine in New York City; Frederick Kaplan, MD, of the Perelman School of Medicine at the University of Pennsylvania; and Cynthia Tifft, MD, PhD, of the National Institutes of Health. The three, along with other honorees, will be recognized for their work to improve the lives of patients affected by rare diseases.

The annual charity event will take place this year on May 18 at the Ronald Reagan Building and International Trade Center Amphitheatre in Washington DC. The event is open to the public and is a fundraiser to support NORD programs and services for all individuals who have rare diseases.

Honorees at NORD’s 2017 Rare Impact Awards will include three clinicians and researchers with expertise in rare diseases. They are Robert Desnick, MD, PhD, of Mount Sinai Hospital and the Icahn School of Medicine in New York City; Frederick Kaplan, MD, of the Perelman School of Medicine at the University of Pennsylvania; and Cynthia Tifft, MD, PhD, of the National Institutes of Health. The three, along with other honorees, will be recognized for their work to improve the lives of patients affected by rare diseases.

The annual charity event will take place this year on May 18 at the Ronald Reagan Building and International Trade Center Amphitheatre in Washington DC. The event is open to the public and is a fundraiser to support NORD programs and services for all individuals who have rare diseases.

To enjoy an evening of inspiration, award presentations, and music, reserve your seat now for the 2017 NORD Rare Impact Awards, which will take place Thursday, May 18, at the Ronald Reagan Building and International Trade Center in Washington, DC.

To enjoy an evening of inspiration, award presentations, and music, reserve your seat now for the 2017 NORD Rare Impact Awards, which will take place Thursday, May 18, at the Ronald Reagan Building and International Trade Center in Washington, DC.

To enjoy an evening of inspiration, award presentations, and music, reserve your seat now for the 2017 NORD Rare Impact Awards, which will take place Thursday, May 18, at the Ronald Reagan Building and International Trade Center in Washington, DC.