Featured News

Living Rare Forum and Rare Impact Awards: Texas-sized success

Over 500 individuals came out for the 2019 Living Rare, Living Stronger NORD Patient & Family Forum featuring the Rare Impact Awards in Houston, TX, June 21-23. The exceptional weekend was filled with learning, bonding and fun! NORD's Living Rare Forum is a traveling event and we are looking forward to visiting a different city in 2020. Stay tuned for details coming soon! For now, check out this recap and photos of this year's unforgettable event.

Registration open: 2019 NORD Rare Summit

The time is now to register for NORD's most highly anticipated event of the year, the Rare Diseases and Orphan Products Breakthrough Summit, taking place October 21-22 in Washington, DC. As technology in rare disease accelerates and with topics like drug pricing taking top priority in government, we must act immediately to drive innovation and collaboration to develop stronger resources, build better outcomes and find cures for the community. In 2019, we believe "The Time is Now" to prioritize action in making positive changes for the millions of Americans living with rare diseases. Register today!

New educational resource available for community

NORD is excited to have launched a new resource featuring videos on rare diseases and related topics for patients, caregivers, students and professionals. NORD's Rare Disease Video Library will grow over time with additional content added on an ongoing basis and resides on www.rarediseases.org, which receives traffic from over 1.3 million visitors per month. Learn more.

Ten new research grant awardees announced

NORD has announced ten new grants in eight diseases states as part of its Rare Disease Research Grant Program, which celebrates its 30th anniversary in 2019. Learn more.

Digital education initiative underway

NORD and PlatfromQ Health are teaming up to create accredited online education for healthcare professionals to ensure they have the information needed to provide the best care possible for the 25-30 million Americans living with rare diseases. Learn more.

June spotlight on Pseudomyxoma Peritonei, Appendix Cancer (ACPMP)

As part of the 30th anniversary of NORD’s Rare Disease Research Grant Program, we launched a year-long campaign called Rare Spotlight to feature a rare disease each month for which NORD has an open research fund to raise awareness and work with the community to raise money for NORD’s research program.

Last month, we put the spotlight on CIDP, a rare malignant growth characterized by the progressive accumulation of mucus-secreting (mucinous) tumor cells within the abdomen and pelvis. Learn more about ACPMP through NORD’s rare disease report on the disorder, in Jenny's story and from NORD member organization, the ACPMP Foundation.

Advocacy News

State policy updates

Wisconsin: The Wisconsin legislature passed Senate Bill 26, legislation to reform step therapy.
Delaware: The Delaware legislature passed, and Governor Carney signed, House Bill 105, legislation to reform step therapy.
New York: The New York legislature passed Senate Bill 4497, legislation to create a rare disease advisory council in the state.
Maine: The Maine legislature passed, and Governor Mills signed, Legislative Document 1009, a bill to reform step therapy.
Indiana: NORD submitted comments to the state in opposition to its waiver regarding its work requirement in Medicaid.
Montana: NORD submitted comments to the state in support of its waiver to implement a reinsurance program.
Texas: NORD sent a letter in support of Senate Bill 747, legislation to improve the state’s newborn screening program, which Governor Abbott then signed.

Federal policy updates

Federally, NORD advocated for the Newborn Screening Saves Lives Act of 2019 (H.R.2507), which had its first hearing in the House Committee on Energy and Commerce last month. NORD also advocated on drug pricing and for legislation to lower health care costs, specifically NORD sent a letter of support for the Lower Health Care Costs Act of 2019 (S.1895). Finally, NORD submitted comments last month to the Centers for Medicare and Medicaid Services regarding the new technology add-on payment program and the need to ensure that systems for reimbursement do not prevent access. For more information on NORD's advocacy, check out our policy communications here.

This is just a sampling! Our volunteer Ambassadors across the country are busy carrying out community events and advocating on a whole range of issues. Check out our Rare Action Center for ways to make your voice heard!

Patient & Caregiver Resources

Upcoming webinar on self-advocacy and care coordination

NORD's RareEDU webinars cover topics important to patients and caregivers. These webinars are free and intended for everyone to access. We are pleased to present our next webinar, which will be on self-advocacy and care coordination, taking place on Wednesday, July 24, 2:00p.m. EDT. Register here.

NORD adds and updates educational rare disease reports

Over the last month, NORD's Educational Initiatives team published 3 new rare disease reports on ADCY5-Related Dyskinesia, Bohring-Opitz Syndrome, and Follicular Lymphoma. In addition, the team updated 3 rare disease reports on Acute Intermittent Porphyria, Bartter Syndrome and Fabry Disease.

Webinar recordings now available

Last month, NORD presented two webinars on generics and biosimilars and specialty pharmacies. The recordings for both webinars are now available on NORD's Youtube channel here.

NORD Member Updates

University of Pittsburgh joins Students for Rare

Last month, University of Pittsburgh joined Students for Rare! The chapter is housed in the Graduate School of Public Health, Department of Human Genetics. It will be led by Erika Dreikorn, Lingling Chen and Justin Dutta. It is advised by Dr. David Finegold.

NORD Member Updates

NORD is pleased to welcome two new patient advocacy groups to
the growing family of NORD Member Organizations.

Cure VCP Disease, Inc. - serving those with diseases related to mutations of the Valosin Containing Protein gene.
FoxG1 Research Foundation - serving those with FOXG1 syndrome.

Member News

American Partnership for Eosinophilic Disorders

Registration is open for APFED’s Patient Education Conference on Eosinophil-Associated Diseases. Eos Connection 2019 will be held in Washington, DC on July 25-27. Learn about the agenda, activities, lodging and registration on their website. More.

CMTC-OVM

Join CMTC-OVM on July 6 for their annual Global Family Day hosted at the Efteling. The event is an all-day affair including hours of fun for the entire family and opportunities to meet other patients and families. More.

Ehlers-Danlos Society

Join the Ehlers-Danlos Society for their 2019 Global Learning Conference on July 30 through August 2 in Nashville, TN. The annual conference will feature presentations and discussion groups led by world-leading experts on the Ehlers-Danlos syndromes (EDS) and hypermobility spectrum disorders (HSD). More.

Fibrous Dysplasia Foundation

Fibrous Dysplasia Foundation is seeking an Executive Director. Check out the job description and apply if you meet the criteria. More.

Glut1 Deficiency Foundation

The Glut1 Deficiency Foundation will be hosting its 8th biennial conference on Glut1 Deficiency on July 11 and 12 in Washington, DC. It’s an opportunity to come together with patients, families and professionals to meet, share and learn in a historic and exciting location. More.

HCU Network America

Join HCU Network America at their 2nd Homocystinuria Conference, Accelerating Towards a Cure, October 19-20 in Indianapolis. More.

International FOP Association

The International Fibrodysplasia Ossificans Progressiva Association (IFOPA) is now accepting applications to fill the position of Registry Project Manager. Visit their website to apply. More.

International Society for Mannisidosis and Related Diseases (ISMRD)

ISMRD is hosting their 6th International conference on Glycoproteinoses in Atlanta July 25-27. This conference will bring scientists, clinicians and families together to share knowledge and inspiration while moving towards the goal of translating basic science discoveries into therapies for the glycoproteinoses. More.

International Waldenstrom's Macroglobulinemia Foundation (IWMF)

IWMF is searching for a Chief Executive Officer to lead the IWMF team to a future with better tomorrows for people worldwide who are affected by Waldenstrom’s macroglobulinemia. More.

Liv4TheCure

Liv4TheCure is spreading awareness about Wolf Hirschhorn Syndrome. Check out Olivia's video and help promote positive change in the rare disease community. More.

Myotonic Dystrophy Foundation

Registration is open for the MDF DM Days in Kansas City, MO and New Haven, CT. These are special one-day events that bring together clinicians, researchers and DM family members to share information and resources and provide close-to-home access to educational sessions More.

National Foundation for Ectodermal Dysplasias

NFED invites everyone affected by ectodermal dysplasias and their loved ones to attend their Family Conference in Chicago July 11-13. This three-day event will provide opportunities to develop a support network, hear the latest research developments and receive answers to pressing questions from medical and dental professionals. More.

Rare Kids Network

Rare Kids Network invites you to donate blood on July 12 in Gainesville, VA to support patients in need of blood, platelets and plasma. Inova Blood Donor Services will provide a gift to all donors. More.

Sofia Sees Hope

Sofia Sees Hope invites you to attend their LCA Family Conference hosted in Philadelphia July 26-28. Attendees will engage in a robust and interactive exchange of knowledge, ideas and viewpoints while making new connections in the Leber congenital amaurosis (LCA) and rare inherited retinal disease (IRD) community. More.

Transverse Myelitis Association

The Transverse Myelitis Association invites you to join their Rare Neuro-Immune Disorders Symposium (RNDS) on September 19. RNDS is dedicated to the exchange of information regarding diagnosis, research and treatment strategies, and to providing an opportunity to bring together the community diagnosed with rare neuro-immune disorders. More.

Vasculitis Foundation

The Vasculitis Foundation encourages all medical professionals to register for the Vasculitis CME course to examine and review current knowledge about the biology and management of vasculitis. The course draws together experts in the fields of epidemiology, imaging and treatment of major forms of vascular inflammatory diseases. More.

VHL Alliance

The VHL Alliance is pleased to announce a call for applications for research grant support on topics relevant to von Hippel-Lindau disease. Projects should further the understanding of VHL disease. Letters of intent should be emailed to research@vhl.org no later than July 15. More.

The XLH Network, Inc.

Registration is open for the XLH Network’s 2019 Community Connections in Denver, CO (7/8), Columbus, OH (9/28), and Birmingham, AL (10/12). The XLH Community Connection Day is a full-day educational and social event that includes patient stories, round table discussions and presentations by local physicians and other clinicians. More.

FDA News

Orphan drug update

June's latest on drug approvals, orphan and breakthrough designations.

Patient Recruitment

Anyone considering participating in a clinical trial should discuss the matter with his or her physician. NORD does not endorse or recommend any particular studies.

For a sampling of current clinical trials, visit the NORD website here.

Myasthenia Gravis

argenx BVBA is conducting a randomized, double-blind, placebo controlled, multicenter phase 3 trial to evaluate the efficacy, safety, tolerability, quality of life and impact on normal daily activities of efgartigimod in patients who have myasthenia gravis with generalized muscle weakness (gMG). This study is enrolling ACHR +, MUSK, LRP4 and seronegative gMG patients. More.

Trichotillomania

Promentis Pharmaceuticals, Inc. is conducting study is to explore the safety, tolerability and activity of SXC-2023 when dosed for 6 weeks versus placebo in adult patients with moderate to severe trichotillomania. More.

Miscellaneous Updates

NORD welcomes new Director of Federal Policy

Michelle Adams has joined NORD as Director of Federal Policy. She brings 20 years of experience in policy, including having worked as a Legislative Assistant and Legislative Director for 2 U.S. Representatives and a U.S. Senator, as Director of Public Policy for the Friends of the Global Fight Against AIDS, Tuberculosis and Malaria and most recently as a Congressional Affairs Specialist for the FDA. Michelle has a Master of Government and Master of Public Health from John Hopkins University.

Save the date for the 2019 Disorder: Rare Disease Film Festival

Disorder: The Rare Disease Film Festival is taking place November 9-10 in San Francisco and will present the largest collection of films about rare diseases to date. Check out the 2019 event trailer here.