News from NORD

The Aplastic Anemia (AA) and Myelodysplastic Syndromes (MDS) International Foundation is accepting registration for three webinars to be offered in August 2017. They are: “MDS Non-Transplant Treatments: What’s in the Pipeline?” on Thursday, August 17; “PNH Clonal Expansion and Prognosis to MDS” on Thursday, August 24; and “MDS Research Updates from ASCO and EHA in 2017” on Tuesday, August 29.

The Aplastic Anemia (AA) and Myelodysplastic Syndromes (MDS) International Foundation is accepting registration for three webinars to be offered in August 2017. They are: “MDS Non-Transplant Treatments: What’s in the Pipeline?” on Thursday, August 17; “PNH Clonal Expansion and Prognosis to MDS” on Thursday, August 24; and “MDS Research Updates from ASCO and EHA in 2017” on Tuesday, August 29.

The Aplastic Anemia (AA) and Myelodysplastic Syndromes (MDS) International Foundation is accepting registration for three webinars to be offered in August 2017. They are: “MDS Non-Transplant Treatments: What’s in the Pipeline?” on Thursday, August 17; “PNH Clonal Expansion and Prognosis to MDS” on Thursday, August 24; and “MDS Research Updates from ASCO and EHA in 2017” on Tuesday, August 29.

An article on the Columbia University Medical Center website describes how neurosurgeon Jeffrey Bruce, MD, helped NORD develop a report for patient/family education on anaplastic astrocytoma. Dr. Bruce is one of many rare disease medical experts who volunteer their time and expertise to help NORD provide information on rare conditions for patients and their families.

An article on the Columbia University Medical Center website describes how neurosurgeon Jeffrey Bruce, MD, helped NORD develop a report for patient/family education on anaplastic astrocytoma. Dr. Bruce is one of many rare disease medical experts who volunteer their time and expertise to help NORD provide information on rare conditions for patients and their families.

An article on the Columbia University Medical Center website describes how neurosurgeon Jeffrey Bruce, MD, helped NORD develop a report for patient/family education on anaplastic astrocytoma. Dr. Bruce is one of many rare disease medical experts who volunteer their time and expertise to help NORD provide information on rare conditions for patients and their families.

Patients with serious or immediately life-threatening diseases or conditions who have no comparable or satisfactory alternative therapy and who seek access to potentially life-saving investigational drugs can use the FDA’s new Expanded Access Navigator to guide them through the process. The Navigator was a team effort led by the Reagan-Udall Foundation in collaboration with patient advocacy groups, the pharmaceutical industry, the FDA, and others in the Federal government.

Patients with serious or immediately life-threatening diseases or conditions who have no comparable or satisfactory alternative therapy and who seek access to potentially life-saving investigational drugs can use the FDA’s new Expanded Access Navigator to guide them through the process. The Navigator was a team effort led by the Reagan-Udall Foundation in collaboration with patient advocacy groups, the pharmaceutical industry, the FDA, and others in the Federal government.

Patients with serious or immediately life-threatening diseases or conditions who have no comparable or satisfactory alternative therapy and who seek access to potentially life-saving investigational drugs can use the FDA’s new Expanded Access Navigator to guide them through the process. The Navigator was a team effort led by the Reagan-Udall Foundation in collaboration with patient advocacy groups, the pharmaceutical industry, the FDA, and others in the Federal government.

Mary Dunkle, NORD Vice President of Educational Initiatives, will speak on a panel discussing the role and needs of caregivers at “The Science of Caregiving: Bringing Voices Together” to be hosted by the National Institute of Nursing Research (NINR) on August 7-8, 2017, in Bethesda, Maryland. This conference is open to all and will also be livestreamed and archived for on-demand viewing. 

Mary Dunkle, NORD Vice President of Educational Initiatives, will speak on a panel discussing the role and needs of caregivers at “The Science of Caregiving: Bringing Voices Together” to be hosted by the National Institute of Nursing Research (NINR) on August 7-8, 2017, in Bethesda, Maryland. This conference is open to all and will also be livestreamed and archived for on-demand viewing. 

Mary Dunkle, NORD Vice President of Educational Initiatives, will speak on a panel discussing the role and needs of caregivers at “The Science of Caregiving: Bringing Voices Together” to be hosted by the National Institute of Nursing Research (NINR) on August 7-8, 2017, in Bethesda, Maryland. This conference is open to all and will also be livestreamed and archived for on-demand viewing. 

Food and Drug Administration (FDA) Commissioner Scott Gottlieb, MD, will present the keynote address in the opening session of the 2017 NORD Summit. Dr. Gottlieb, who was sworn in as FDA Commissioner in May 2017, previously served as FDA’s Deputy Commissioner for Medical and Scientific Affairs. Before that, he was Senior Adviser to the FDA Commissioner.

Mike Porath, award-winning journalist and founder/CEO of The Mighty, will present the patient keynote. He has held writing, editing, and executive positions at ABC News, NBC News, the New York Times, and AOL. Mr. Porath is the father of a child with Dup15q syndrome and sits on the board of Dup15q Alliance.

Food and Drug Administration (FDA) Commissioner Scott Gottlieb, MD, will present the keynote address in the opening session of the 2017 NORD Summit. Dr. Gottlieb, who was sworn in as FDA Commissioner in May 2017, previously served as FDA’s Deputy Commissioner for Medical and Scientific Affairs. Before that, he was Senior Adviser to the FDA Commissioner.

Mike Porath, award-winning journalist and founder/CEO of The Mighty, will present the patient keynote. He has held writing, editing, and executive positions at ABC News, NBC News, the New York Times, and AOL. Mr. Porath is the father of a child with Dup15q syndrome and sits on the board of Dup15q Alliance.

Food and Drug Administration (FDA) Commissioner Scott Gottlieb, MD, will present the keynote address in the opening session of the 2017 NORD Summit. Dr. Gottlieb, who was sworn in as FDA Commissioner in May 2017, previously served as FDA’s Deputy Commissioner for Medical and Scientific Affairs. Before that, he was Senior Adviser to the FDA Commissioner.

Mike Porath, award-winning journalist and founder/CEO of The Mighty, will present the patient keynote. He has held writing, editing, and executive positions at ABC News, NBC News, the New York Times, and AOL. Mr. Porath is the father of a child with Dup15q syndrome and sits on the board of Dup15q Alliance.

Promoting Earlier Diagnosis, the Power of Data Sharing, Next-Generation Treatments, and Advancing Clinical Trials will be among the topics discussed at the 2017 NORD Rare Diseases and Orphan Products Breakthrough Summit, to be held on October 16-17, 2017, in Washington DC. The program is open to all and will feature more than 80 expert speakers. Learn more and download the agenda. 

Register by August 25 for Early-Bird Pricing

Online registration is now open for the NORD Summit. Register by August 25 to save up to $400. The NORD Summit will take place October 16-17 at the Marriott Wardman Park Hotel in Washington DC. 

Submit a Poster Abstract or Become an Exhibitor or Sponsor

August 18 is the deadline to submit abstracts for the Summit Poster Session. The overall poster theme is “Life-Transforming Treatments.” In addition, there are opportunities to exhibit at the Summit or become a sponsor. Exhibiting and sponsorship inquiries may be sent to amoore@rarediseases.org.  

Promoting Earlier Diagnosis, the Power of Data Sharing, Next-Generation Treatments, and Advancing Clinical Trials will be among the topics discussed at the 2017 NORD Rare Diseases and Orphan Products Breakthrough Summit, to be held on October 16-17, 2017, in Washington DC. The program is open to all and will feature more than 80 expert speakers. Learn more and download the agenda. 

Register by August 25 for Early-Bird Pricing

Online registration is now open for the NORD Summit. Register by August 25 to save up to $400. The NORD Summit will take place October 16-17 at the Marriott Wardman Park Hotel in Washington DC. 

Submit a Poster Abstract or Become an Exhibitor or Sponsor

August 18 is the deadline to submit abstracts for the Summit Poster Session. The overall poster theme is “Life-Transforming Treatments.” In addition, there are opportunities to exhibit at the Summit or become a sponsor. Exhibiting and sponsorship inquiries may be sent to amoore@rarediseases.org.  

Promoting Earlier Diagnosis, the Power of Data Sharing, Next-Generation Treatments, and Advancing Clinical Trials will be among the topics discussed at the 2017 NORD Rare Diseases and Orphan Products Breakthrough Summit, to be held on October 16-17, 2017, in Washington DC. The program is open to all and will feature more than 80 expert speakers. Learn more and download the agenda. 

Register by August 25 for Early-Bird Pricing

Online registration is now open for the NORD Summit. Register by August 25 to save up to $400. The NORD Summit will take place October 16-17 at the Marriott Wardman Park Hotel in Washington DC. 

Submit a Poster Abstract or Become an Exhibitor or Sponsor

August 18 is the deadline to submit abstracts for the Summit Poster Session. The overall poster theme is “Life-Transforming Treatments.” In addition, there are opportunities to exhibit at the Summit or become a sponsor. Exhibiting and sponsorship inquiries may be sent to amoore@rarediseases.org.  

Public health professionals, clinicians, and researchers are invited to a free webinar being hosted by CDC Division of Blood Disorders on “Women and Girls with Bleeding Disorders: Challenges in Diagnosis and Management.” The webinar will take place on May 18, 2017, from 2 pm to 3 pm. Register online.

Public health professionals, clinicians, and researchers are invited to a free webinar being hosted by CDC Division of Blood Disorders on “Women and Girls with Bleeding Disorders: Challenges in Diagnosis and Management.” The webinar will take place on May 18, 2017, from 2 pm to 3 pm. Register online.

Public health professionals, clinicians, and researchers are invited to a free webinar being hosted by CDC Division of Blood Disorders on “Women and Girls with Bleeding Disorders: Challenges in Diagnosis and Management.” The webinar will take place on May 18, 2017, from 2 pm to 3 pm. Register online.

The Transverse Myelitis Association has announced that registration is now open for the 2017 Rare Neuro-Immune Disorders Symposium to take place October 20-21, 2017, in Columbus, Ohio. This education and advocacy conference is for individuals, families, and caregivers affected by acute disseminated encephalomyelitis (ADEM), neuromyelitis optica spectrum disorder (NMOSD), optic neuritis (ON), and transverse myelitis including the subtype acute flaccid myelitis.

The Transverse Myelitis Association has announced that registration is now open for the 2017 Rare Neuro-Immune Disorders Symposium to take place October 20-21, 2017, in Columbus, Ohio. This education and advocacy conference is for individuals, families, and caregivers affected by acute disseminated encephalomyelitis (ADEM), neuromyelitis optica spectrum disorder (NMOSD), optic neuritis (ON), and transverse myelitis including the subtype acute flaccid myelitis.

The Transverse Myelitis Association has announced that registration is now open for the 2017 Rare Neuro-Immune Disorders Symposium to take place October 20-21, 2017, in Columbus, Ohio. This education and advocacy conference is for individuals, families, and caregivers affected by acute disseminated encephalomyelitis (ADEM), neuromyelitis optica spectrum disorder (NMOSD), optic neuritis (ON), and transverse myelitis including the subtype acute flaccid myelitis.

The Spinal CSF Leak Foundation will host an Intracranial Hypotension Symposium on October 14, 2017, in Santa Monica, California, in partnership with Cedars-Sinai. Registration is now open.

The Spinal CSF Leak Foundation will host an Intracranial Hypotension Symposium on October 14, 2017, in Santa Monica, California, in partnership with Cedars-Sinai. Registration is now open.

The Spinal CSF Leak Foundation will host an Intracranial Hypotension Symposium on October 14, 2017, in Santa Monica, California, in partnership with Cedars-Sinai. Registration is now open.

On May 20, 2017, in Washington DC, the Pulmonary Hypertension Association (PHA) and the American Thoracic Society will host a program in which participants can learn from leading pulmonary hypertension experts and meet other patients, families and caregivers. The event is part of a Meet the Experts session hosted by PHA and the American Thoracic Society’s Public Advisory Roundtable.

On May 20, 2017, in Washington DC, the Pulmonary Hypertension Association (PHA) and the American Thoracic Society will host a program in which participants can learn from leading pulmonary hypertension experts and meet other patients, families and caregivers. The event is part of a Meet the Experts session hosted by PHA and the American Thoracic Society’s Public Advisory Roundtable.

On May 20, 2017, in Washington DC, the Pulmonary Hypertension Association (PHA) and the American Thoracic Society will host a program in which participants can learn from leading pulmonary hypertension experts and meet other patients, families and caregivers. The event is part of a Meet the Experts session hosted by PHA and the American Thoracic Society’s Public Advisory Roundtable.