News from NORD

NORD’s Research Grant Program is still accepting proposals for the study of three rare diseases: cat eye syndrome, malonic aciduria, and post-orgasmic illness syndrome. All interested US and international researchers are encouraged to apply. Learn more.

NORD’s Research Grant Program is still accepting proposals for the study of three rare diseases: cat eye syndrome, malonic aciduria, and post-orgasmic illness syndrome. All interested US and international researchers are encouraged to apply. Learn more.

NORD’s Research Grant Program is still accepting proposals for the study of three rare diseases: cat eye syndrome, malonic aciduria, and post-orgasmic illness syndrome. All interested US and international researchers are encouraged to apply. Learn more.

In January of 1983, President Ronald Reagan signed the Orphan Drug Act, launching a new era of hope for the millions of Americans with diseases so rare that no pharmaceutical company was pursuing development of treatments. A few months later, the patient advocates who had worked together to get that law enacted formally announced their collaboration as the National Organization for Rare Disorders (NORD), to provide advocacy, education, research, and patient services on behalf of all people affected by rare diseases. Throughout 2018, NORD and others in the rare disease community will be celebrating this 35th anniversary year. While only a dozen rare disease treatments had been developed by industry in the decade before 1983, more than 500 have been approved by FDA since then and many more are in the pipeline. Many of these are breakthrough therapies that have been life-saving, or have significantly improved quality of life, for patients who previously had no therapy.  View archived video from 30th anniversary about the role of patient advocates in enactment of the Orphan Drug Act.

In January of 1983, President Ronald Reagan signed the Orphan Drug Act, launching a new era of hope for the millions of Americans with diseases so rare that no pharmaceutical company was pursuing development of treatments. A few months later, the patient advocates who had worked together to get that law enacted formally announced their collaboration as the National Organization for Rare Disorders (NORD), to provide advocacy, education, research, and patient services on behalf of all people affected by rare diseases. Throughout 2018, NORD and others in the rare disease community will be celebrating this 35th anniversary year. While only a dozen rare disease treatments had been developed by industry in the decade before 1983, more than 500 have been approved by FDA since then and many more are in the pipeline. Many of these are breakthrough therapies that have been life-saving, or have significantly improved quality of life, for patients who previously had no therapy.  View archived video from 30th anniversary about the role of patient advocates in enactment of the Orphan Drug Act.

In January of 1983, President Ronald Reagan signed the Orphan Drug Act, launching a new era of hope for the millions of Americans with diseases so rare that no pharmaceutical company was pursuing development of treatments. A few months later, the patient advocates who had worked together to get that law enacted formally announced their collaboration as the National Organization for Rare Disorders (NORD), to provide advocacy, education, research, and patient services on behalf of all people affected by rare diseases. Throughout 2018, NORD and others in the rare disease community will be celebrating this 35th anniversary year. While only a dozen rare disease treatments had been developed by industry in the decade before 1983, more than 500 have been approved by FDA since then and many more are in the pipeline. Many of these are breakthrough therapies that have been life-saving, or have significantly improved quality of life, for patients who previously had no therapy.  View archived video from 30th anniversary about the role of patient advocates in enactment of the Orphan Drug Act.

On Thursday, May 17, 2018, NORD will honor clinicians, researchers, patient advocates, and others who have made outstanding contributions to improving the lives of people with rare diseases. This will take place at the Rare Impact Awards event, which takes place each year at this time in Washington, DC. This year, the venue will be the Andrew W. Mellon Auditorium. Individuals being honored include Robert Campbell, MD, of Children’s Hospital of Philadelphia, who is receiving a Lifetime Achievement Award; Richard Pazdur, MD, of the FDA, who is receiving the Public Health Leadership Award; and Elisabeth Dykens, PhD, of Vanderbilt University, who is being honored for her research related to rare genetic syndromes. Read about all the honorees.

The Rare Impact Awards Celebration is open to the public. Registration is open now on the NORD website.

On Thursday, May 17, 2018, NORD will honor clinicians, researchers, patient advocates, and others who have made outstanding contributions to improving the lives of people with rare diseases. This will take place at the Rare Impact Awards event, which takes place each year at this time in Washington, DC. This year, the venue will be the Andrew W. Mellon Auditorium. Individuals being honored include Robert Campbell, MD, of Children’s Hospital of Philadelphia, who is receiving a Lifetime Achievement Award; Richard Pazdur, MD, of the FDA, who is receiving the Public Health Leadership Award; and Elisabeth Dykens, PhD, of Vanderbilt University, who is being honored for her research related to rare genetic syndromes. Read about all the honorees.

The Rare Impact Awards Celebration is open to the public. Registration is open now on the NORD website.

On Thursday, May 17, 2018, NORD will honor clinicians, researchers, patient advocates, and others who have made outstanding contributions to improving the lives of people with rare diseases. This will take place at the Rare Impact Awards event, which takes place each year at this time in Washington, DC. This year, the venue will be the Andrew W. Mellon Auditorium. Individuals being honored include Robert Campbell, MD, of Children’s Hospital of Philadelphia, who is receiving a Lifetime Achievement Award; Richard Pazdur, MD, of the FDA, who is receiving the Public Health Leadership Award; and Elisabeth Dykens, PhD, of Vanderbilt University, who is being honored for her research related to rare genetic syndromes. Read about all the honorees.

The Rare Impact Awards Celebration is open to the public. Registration is open now on the NORD website.

The OIF will host its biennial national conference in Baltimore July 13-15, 2018,. The program will feature sessions on medical care and practical living, forums with leading experts in osteogenesis imperfect care and research, and other opportunities. More.  

The OIF will host its biennial national conference in Baltimore July 13-15, 2018,. The program will feature sessions on medical care and practical living, forums with leading experts in osteogenesis imperfect care and research, and other opportunities. More.  

The OIF will host its biennial national conference in Baltimore July 13-15, 2018,. The program will feature sessions on medical care and practical living, forums with leading experts in osteogenesis imperfect care and research, and other opportunities. More.  

Register now for the inaugural Speaking of NEC: Unplugged on June 11, 2018, in Cromwell Connecticut, a one-day regional conference focused on reducing the devastating effects of necrotizing enterocolitis (NEC) on premature infants and their families. More. 

Register now for the inaugural Speaking of NEC: Unplugged on June 11, 2018, in Cromwell Connecticut, a one-day regional conference focused on reducing the devastating effects of necrotizing enterocolitis (NEC) on premature infants and their families. More. 

Register now for the inaugural Speaking of NEC: Unplugged on June 11, 2018, in Cromwell Connecticut, a one-day regional conference focused on reducing the devastating effects of necrotizing enterocolitis (NEC) on premature infants and their families. More. 

Join the MDS Foundation for a Patient and Family/Caregiver forum on May 5, 2018, in Salt Lake City. This one-day forum provides new patients, long-term survivors, and caregivers the opportunity to learn from experts about treatments and strategies for living with MDS. More.

Join the MDS Foundation for a Patient and Family/Caregiver forum on May 5, 2018, in Salt Lake City. This one-day forum provides new patients, long-term survivors, and caregivers the opportunity to learn from experts about treatments and strategies for living with MDS. More.

Join the MDS Foundation for a Patient and Family/Caregiver forum on May 5, 2018, in Salt Lake City. This one-day forum provides new patients, long-term survivors, and caregivers the opportunity to learn from experts about treatments and strategies for living with MDS. More.

In May 2018, the IPPF will bring together pemphigus and pemphigoid patients, clinicians, researchers, and industry partners to focus on ongoing or future clinical trials and their underlying science. This will take place prior to the International Investigative Dermatology meeting in Orlando. More.

In May 2018, the IPPF will bring together pemphigus and pemphigoid patients, clinicians, researchers, and industry partners to focus on ongoing or future clinical trials and their underlying science. This will take place prior to the International Investigative Dermatology meeting in Orlando. More.

In May 2018, the IPPF will bring together pemphigus and pemphigoid patients, clinicians, researchers, and industry partners to focus on ongoing or future clinical trials and their underlying science. This will take place prior to the International Investigative Dermatology meeting in Orlando. More.

The Ehlers-Danlos Society will hold an International Symposium Sept. 26-29, 2018, in Ghent, Belgium. Clinicians and researchers are encouraged to attend to discuss the molecular, pathogenic, clinical, and management aspects of all types of Ehlers-Danlos syndromes. More.

The Ehlers-Danlos Society will hold an International Symposium Sept. 26-29, 2018, in Ghent, Belgium. Clinicians and researchers are encouraged to attend to discuss the molecular, pathogenic, clinical, and management aspects of all types of Ehlers-Danlos syndromes. More.

The Ehlers-Danlos Society will hold an International Symposium Sept. 26-29, 2018, in Ghent, Belgium. Clinicians and researchers are encouraged to attend to discuss the molecular, pathogenic, clinical, and management aspects of all types of Ehlers-Danlos syndromes. More.

Join the Dup 15q Alliance and the Angelman Syndrome Research Foundation August 6-7, 2018, for world-class scientific, translational, and clinical presentations. This symposium allows for the sharing of unpublished work, which leads to conceptual discussions and helps to accelerate therapeutic opportunities for both disorders. More.

Join the Dup 15q Alliance and the Angelman Syndrome Research Foundation August 6-7, 2018, for world-class scientific, translational, and clinical presentations. This symposium allows for the sharing of unpublished work, which leads to conceptual discussions and helps to accelerate therapeutic opportunities for both disorders. More.

Join the Dup 15q Alliance and the Angelman Syndrome Research Foundation August 6-7, 2018, for world-class scientific, translational, and clinical presentations. This symposium allows for the sharing of unpublished work, which leads to conceptual discussions and helps to accelerate therapeutic opportunities for both disorders. More.

Cure SMA is conducting a survey of pediatricians to assess baseline awareness regarding spinal muscular atrophy. Support Cure SMA and take the survey! More.

Cure SMA is conducting a survey of pediatricians to assess baseline awareness regarding spinal muscular atrophy. Support Cure SMA and take the survey! More.

Cure SMA is conducting a survey of pediatricians to assess baseline awareness regarding spinal muscular atrophy. Support Cure SMA and take the survey! More.