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Free Genetic Testing Program Is Launched for Patients With Inherited Retinal Diseases
A free genetic testing program for eligible patients living with inherited retinal diseases has been developed in consultation with patient advocacy groups in the retinal disease community and has now been launched. Full program information is available here.
A free genetic testing program for eligible patients living with inherited retinal diseases has been developed in consultation with patient advocacy groups in the retinal disease community and has now been launched. Full program information is available here.
A free genetic testing program for eligible patients living with inherited retinal diseases has been developed in consultation with patient advocacy groups in the retinal disease community and has now been launched. Full program information is available here.
FDA Awards 21 Grants for Rare Disease Product Development
New clinical research grants totaling more than $23 million have been awarded to support the development of diagnostics and treatments for patients with rare disease. More than 40% of the awards focus on treatments for children. The grants were awarded to investigators from academia and industry in the US and abroad through the FDA’s Office of Orphan Product Development.
New clinical research grants totaling more than $23 million have been awarded to support the development of diagnostics and treatments for patients with rare disease. More than 40% of the awards focus on treatments for children. The grants were awarded to investigators from academia and industry in the US and abroad through the FDA’s Office of Orphan Product Development.
New clinical research grants totaling more than $23 million have been awarded to support the development of diagnostics and treatments for patients with rare disease. More than 40% of the awards focus on treatments for children. The grants were awarded to investigators from academia and industry in the US and abroad through the FDA’s Office of Orphan Product Development.
NORD Urges Congress to Pass 21st Century Cures Act
The National Organization for Rare Disorders (NORD) is urging Congress to pass the 21st Century Cures Act, which includes provisions important to the rare disease community such as additional funding for NIH and continuation of the FDA Patient-Focused Drug Development Initiative.
This legislation also would reauthorize the Rare Pediatric Disease Priority Review Voucher Program, which incentivizes development of treatments for rare pediatric diseases. Through this program, priority review vouchers are awarded to companies that develop new treatments for children with rare diseases. The program will expire at the end of this year, and NORD is advocating its long-term reauthorization.
Watch the NORD website for opportunities to join NORD and its policy partners in supporting this legislation and reauthorization of the voucher program.
The National Organization for Rare Disorders (NORD) is urging Congress to pass the 21st Century Cures Act, which includes provisions important to the rare disease community such as additional funding for NIH and continuation of the FDA Patient-Focused Drug Development Initiative.
This legislation also would reauthorize the Rare Pediatric Disease Priority Review Voucher Program, which incentivizes development of treatments for rare pediatric diseases. Through this program, priority review vouchers are awarded to companies that develop new treatments for children with rare diseases. The program will expire at the end of this year, and NORD is advocating its long-term reauthorization.
Watch the NORD website for opportunities to join NORD and its policy partners in supporting this legislation and reauthorization of the voucher program.
The National Organization for Rare Disorders (NORD) is urging Congress to pass the 21st Century Cures Act, which includes provisions important to the rare disease community such as additional funding for NIH and continuation of the FDA Patient-Focused Drug Development Initiative.
This legislation also would reauthorize the Rare Pediatric Disease Priority Review Voucher Program, which incentivizes development of treatments for rare pediatric diseases. Through this program, priority review vouchers are awarded to companies that develop new treatments for children with rare diseases. The program will expire at the end of this year, and NORD is advocating its long-term reauthorization.
Watch the NORD website for opportunities to join NORD and its policy partners in supporting this legislation and reauthorization of the voucher program.
Nominate a Colleague for a NORD 2017 Rare Impact Award
Continuing a 31-year tradition, the National Organization for Rare Disorders (NORD) will honor medical researchers, clinicians, patients, caregivers, and others who have had a positive impact on the rare disease community at its annual Rare Impact Awards celebration in May 2017. Nominations are now open and may be submitted online. Friday, Jan. 13, is the deadline for submitting nominations.
Nominees can include individuals or organizations. Past honorees have been recognized for their work in advocacy, research, patient care, awareness, ethics, and public policy. The 2017 honorees will be determined by a nominations committee comprised of NORD’s Scientific and Medical Advisory Committee, Board of Directors, and advocates.
The awards ceremony will take place on May 18 in the amphitheater of the Ronald Reagan Building and International Trade Center in Washington, DC. The 2017 Rare Impact Awards will continue NORD’s longstanding tradition of bringing together medical professionals, patients, caregivers, federal officials, and others to celebrate those who have had a positive impact on the community.
Continuing a 31-year tradition, the National Organization for Rare Disorders (NORD) will honor medical researchers, clinicians, patients, caregivers, and others who have had a positive impact on the rare disease community at its annual Rare Impact Awards celebration in May 2017. Nominations are now open and may be submitted online. Friday, Jan. 13, is the deadline for submitting nominations.
Nominees can include individuals or organizations. Past honorees have been recognized for their work in advocacy, research, patient care, awareness, ethics, and public policy. The 2017 honorees will be determined by a nominations committee comprised of NORD’s Scientific and Medical Advisory Committee, Board of Directors, and advocates.
The awards ceremony will take place on May 18 in the amphitheater of the Ronald Reagan Building and International Trade Center in Washington, DC. The 2017 Rare Impact Awards will continue NORD’s longstanding tradition of bringing together medical professionals, patients, caregivers, federal officials, and others to celebrate those who have had a positive impact on the community.
Continuing a 31-year tradition, the National Organization for Rare Disorders (NORD) will honor medical researchers, clinicians, patients, caregivers, and others who have had a positive impact on the rare disease community at its annual Rare Impact Awards celebration in May 2017. Nominations are now open and may be submitted online. Friday, Jan. 13, is the deadline for submitting nominations.
Nominees can include individuals or organizations. Past honorees have been recognized for their work in advocacy, research, patient care, awareness, ethics, and public policy. The 2017 honorees will be determined by a nominations committee comprised of NORD’s Scientific and Medical Advisory Committee, Board of Directors, and advocates.
The awards ceremony will take place on May 18 in the amphitheater of the Ronald Reagan Building and International Trade Center in Washington, DC. The 2017 Rare Impact Awards will continue NORD’s longstanding tradition of bringing together medical professionals, patients, caregivers, federal officials, and others to celebrate those who have had a positive impact on the community.
Certification in Rare Neuroimmunologic Disorders
The Consortium of Multiple Sclerosis Centers (CMSC) has announced that the next Certification Examination in Rare Neuroimmunologic Disorders will take place in January 2017. The application deadline is December 1, 2016. Certification in Rare Neuroimmunologic Disorders (CRND) is intended to validate quality and consistent care to those living with rare neuroimmunologic disorders of the central nervous system.
These rare disorders include transverse myelitis, neuromyelitis optica spectrum disorder, optic neuritis, acute disseminated encephalomyelitis, and anti-NMDA receptor encephalitis. All licensed health professionals with a recommended one year of experience in providing services for patients with rare neuroimmunologic disorders are eligible to take the examination.
Those who successfully pass the certification exam will be eligible to use the registered designation CRND after their names and will receive certificates from CMSC.
Additional information about this certification program and examination may be found on the following page on the CMSC website: http://www.mscare.org/page/about_crnd
The Consortium of Multiple Sclerosis Centers (CMSC) has announced that the next Certification Examination in Rare Neuroimmunologic Disorders will take place in January 2017. The application deadline is December 1, 2016. Certification in Rare Neuroimmunologic Disorders (CRND) is intended to validate quality and consistent care to those living with rare neuroimmunologic disorders of the central nervous system.
These rare disorders include transverse myelitis, neuromyelitis optica spectrum disorder, optic neuritis, acute disseminated encephalomyelitis, and anti-NMDA receptor encephalitis. All licensed health professionals with a recommended one year of experience in providing services for patients with rare neuroimmunologic disorders are eligible to take the examination.
Those who successfully pass the certification exam will be eligible to use the registered designation CRND after their names and will receive certificates from CMSC.
Additional information about this certification program and examination may be found on the following page on the CMSC website: http://www.mscare.org/page/about_crnd
The Consortium of Multiple Sclerosis Centers (CMSC) has announced that the next Certification Examination in Rare Neuroimmunologic Disorders will take place in January 2017. The application deadline is December 1, 2016. Certification in Rare Neuroimmunologic Disorders (CRND) is intended to validate quality and consistent care to those living with rare neuroimmunologic disorders of the central nervous system.
These rare disorders include transverse myelitis, neuromyelitis optica spectrum disorder, optic neuritis, acute disseminated encephalomyelitis, and anti-NMDA receptor encephalitis. All licensed health professionals with a recommended one year of experience in providing services for patients with rare neuroimmunologic disorders are eligible to take the examination.
Those who successfully pass the certification exam will be eligible to use the registered designation CRND after their names and will receive certificates from CMSC.
Additional information about this certification program and examination may be found on the following page on the CMSC website: http://www.mscare.org/page/about_crnd
AT Children’s Project Advisors Elected to National Academy of Sciences
Longtime scientific advisors to the AT (Ataxia Telangiectasia) Children’s Project Nathaniel Heintz, PhD, and Michael Kastan, MD, PhD, have been elected to the National Academy of Sciences.
Longtime scientific advisors to the AT (Ataxia Telangiectasia) Children’s Project Nathaniel Heintz, PhD, and Michael Kastan, MD, PhD, have been elected to the National Academy of Sciences.
Longtime scientific advisors to the AT (Ataxia Telangiectasia) Children’s Project Nathaniel Heintz, PhD, and Michael Kastan, MD, PhD, have been elected to the National Academy of Sciences.
Penn State Graduate Student Hopes to Help Families Find NORD at Time of Rare Disease Diagnosis
Kerri Nelson, a graduate student in the Department of Nursing at Pennsylvania State University and mother of a child with a rare disorder, is conducting a survey with NORD to learn how patients and families find rare disease information that is helpful to them.
“When I began my Doctorate of Nursing Practice, I found the National Organization for Rare Disorders (NORD) and felt that this organization would have been beneficial two years prior when my son was diagnosed with a rare disorder,” she wrote on the NORD blog. As a result, Ms. Nelson got the idea to help those with newly diagnosed children find the NORD website. She has designed a toolkit to be distributed to patients and families at Penn State’s Milton S. Hershey Medical Center. She also developed a brief survey that is posted on the NORD website.
Kerri Nelson, a graduate student in the Department of Nursing at Pennsylvania State University and mother of a child with a rare disorder, is conducting a survey with NORD to learn how patients and families find rare disease information that is helpful to them.
“When I began my Doctorate of Nursing Practice, I found the National Organization for Rare Disorders (NORD) and felt that this organization would have been beneficial two years prior when my son was diagnosed with a rare disorder,” she wrote on the NORD blog. As a result, Ms. Nelson got the idea to help those with newly diagnosed children find the NORD website. She has designed a toolkit to be distributed to patients and families at Penn State’s Milton S. Hershey Medical Center. She also developed a brief survey that is posted on the NORD website.
Kerri Nelson, a graduate student in the Department of Nursing at Pennsylvania State University and mother of a child with a rare disorder, is conducting a survey with NORD to learn how patients and families find rare disease information that is helpful to them.
“When I began my Doctorate of Nursing Practice, I found the National Organization for Rare Disorders (NORD) and felt that this organization would have been beneficial two years prior when my son was diagnosed with a rare disorder,” she wrote on the NORD blog. As a result, Ms. Nelson got the idea to help those with newly diagnosed children find the NORD website. She has designed a toolkit to be distributed to patients and families at Penn State’s Milton S. Hershey Medical Center. She also developed a brief survey that is posted on the NORD website.
NIH Issues Final Rule for Registering Clinical Trials and Reporting Results
The National Institutes of Health (NIH) has published a new policy to assure that results of clinical trials are widely shared. NIH Director Francis Collins, MD, said the final rule and NIH policy that have been issued “will help maximize the value of clinical trials, whether publicly or privately supported, and help us honor our commitments to trial participants, who do so much to help society advance knowledge and improve health.”
The National Institutes of Health (NIH) has published a new policy to assure that results of clinical trials are widely shared. NIH Director Francis Collins, MD, said the final rule and NIH policy that have been issued “will help maximize the value of clinical trials, whether publicly or privately supported, and help us honor our commitments to trial participants, who do so much to help society advance knowledge and improve health.”
The National Institutes of Health (NIH) has published a new policy to assure that results of clinical trials are widely shared. NIH Director Francis Collins, MD, said the final rule and NIH policy that have been issued “will help maximize the value of clinical trials, whether publicly or privately supported, and help us honor our commitments to trial participants, who do so much to help society advance knowledge and improve health.”
October 21 Is Deadline for NORD Research Grant Abstracts
The deadline has been extended to October 21 to submit letters of intent and abstracts for National Organization for Rare Disorders (NORD) research grants for studies related to the following rare diseases:
- Alveolar capillary dysplasia with misalignment of the pulmonary veins (ACD/MPV)
- Appendix cancer and pseudomyxoma peritonei (PMP)
- Autoimmune polyglandular syndrome type 1 (APS Type 1)
- Homocystinuria due to cystathionine beta-synthase deficiency
- Malonic aciduria
- Stiff person syndrome
The NORD Research Grant Program encourages meritorious scientific and clinical research toward new diagnostics, treatments, and/or cures for rare diseases. NORD’s program provides seed grants to clinicians and academic scientists for translational or clinical studies. Data generated in these studies may then be used by researchers to obtain additional funding from government or industry sponsors. Two of NORD’s previous research grants have resulted in FDA-approved orphan products to treat rare diseases. Submissions should be sent to research@rarediseases.org. Click here to view each Request for Proposal in full.
The deadline has been extended to October 21 to submit letters of intent and abstracts for National Organization for Rare Disorders (NORD) research grants for studies related to the following rare diseases:
- Alveolar capillary dysplasia with misalignment of the pulmonary veins (ACD/MPV)
- Appendix cancer and pseudomyxoma peritonei (PMP)
- Autoimmune polyglandular syndrome type 1 (APS Type 1)
- Homocystinuria due to cystathionine beta-synthase deficiency
- Malonic aciduria
- Stiff person syndrome
The NORD Research Grant Program encourages meritorious scientific and clinical research toward new diagnostics, treatments, and/or cures for rare diseases. NORD’s program provides seed grants to clinicians and academic scientists for translational or clinical studies. Data generated in these studies may then be used by researchers to obtain additional funding from government or industry sponsors. Two of NORD’s previous research grants have resulted in FDA-approved orphan products to treat rare diseases. Submissions should be sent to research@rarediseases.org. Click here to view each Request for Proposal in full.
The deadline has been extended to October 21 to submit letters of intent and abstracts for National Organization for Rare Disorders (NORD) research grants for studies related to the following rare diseases:
- Alveolar capillary dysplasia with misalignment of the pulmonary veins (ACD/MPV)
- Appendix cancer and pseudomyxoma peritonei (PMP)
- Autoimmune polyglandular syndrome type 1 (APS Type 1)
- Homocystinuria due to cystathionine beta-synthase deficiency
- Malonic aciduria
- Stiff person syndrome
The NORD Research Grant Program encourages meritorious scientific and clinical research toward new diagnostics, treatments, and/or cures for rare diseases. NORD’s program provides seed grants to clinicians and academic scientists for translational or clinical studies. Data generated in these studies may then be used by researchers to obtain additional funding from government or industry sponsors. Two of NORD’s previous research grants have resulted in FDA-approved orphan products to treat rare diseases. Submissions should be sent to research@rarediseases.org. Click here to view each Request for Proposal in full.
It’s Not Too Late to Register for the NORD Summit
Food and Drug Administration (FDA) Commissioner Robert Califf, MD, will deliver a keynote address on the opening morning of the NORD Rare Diseases and Orphan Products Breakthrough Summit to take place Oct. 17 and 18 in Arlington Virginia. Approximately one-third of the novel new drugs approved by FDA in recent years have been “orphan” drugs for rare diseases.
Dr. Califf will be joined on the program agenda by more than 25 additional FDA speakers, including Janet Woodcock, MD, Director of FDA’s Center for Drug Evaluation and Research, and Peter Marks, MD, PhD, Director of FDA’s Center for Biologics Evaluation and Research.
On Day Two of the conference, Kate Rawson, Senior Editor at Prevision Policy, will provide the morning keynote with a look ahead at possible implications of the national election for the rare disease community. The NORD Summit attracts medical professionals, patient advocates, and others to examine issues related to rare disease research, diagnosis, treatment, and patient access to care. The conference is open to all. Click here to view the agenda.
Food and Drug Administration (FDA) Commissioner Robert Califf, MD, will deliver a keynote address on the opening morning of the NORD Rare Diseases and Orphan Products Breakthrough Summit to take place Oct. 17 and 18 in Arlington Virginia. Approximately one-third of the novel new drugs approved by FDA in recent years have been “orphan” drugs for rare diseases.
Dr. Califf will be joined on the program agenda by more than 25 additional FDA speakers, including Janet Woodcock, MD, Director of FDA’s Center for Drug Evaluation and Research, and Peter Marks, MD, PhD, Director of FDA’s Center for Biologics Evaluation and Research.
On Day Two of the conference, Kate Rawson, Senior Editor at Prevision Policy, will provide the morning keynote with a look ahead at possible implications of the national election for the rare disease community. The NORD Summit attracts medical professionals, patient advocates, and others to examine issues related to rare disease research, diagnosis, treatment, and patient access to care. The conference is open to all. Click here to view the agenda.
Food and Drug Administration (FDA) Commissioner Robert Califf, MD, will deliver a keynote address on the opening morning of the NORD Rare Diseases and Orphan Products Breakthrough Summit to take place Oct. 17 and 18 in Arlington Virginia. Approximately one-third of the novel new drugs approved by FDA in recent years have been “orphan” drugs for rare diseases.
Dr. Califf will be joined on the program agenda by more than 25 additional FDA speakers, including Janet Woodcock, MD, Director of FDA’s Center for Drug Evaluation and Research, and Peter Marks, MD, PhD, Director of FDA’s Center for Biologics Evaluation and Research.
On Day Two of the conference, Kate Rawson, Senior Editor at Prevision Policy, will provide the morning keynote with a look ahead at possible implications of the national election for the rare disease community. The NORD Summit attracts medical professionals, patient advocates, and others to examine issues related to rare disease research, diagnosis, treatment, and patient access to care. The conference is open to all. Click here to view the agenda.