Register Online Now for Rare Disease Investigators' Training Course

FDA and NIH, in collaboration with NORD and Duke University, will offer a three-day course on conducting research on rare diseases and orphan products Oct. 18-20 in Rockville, MD. The course will focus on FDA's regulatory requirements and the clinical trial issues that are especially relevant to rare disease research. Read the flyer.

Register online

House of Representatives
Honors Dr. Campbell

On July 1, a resolution (H.Res. 1499) honoring the achievements of Robert Campbell, MD, was approved by the U.S. House of Representatives. It was sponsored by Congresswoman Debbie Wasserman Schultz (D-FL). Dr. Campbell, of the Children's Hospital of Philadelphia, invented a device known as the expandable titanium rib that has saved the lives of hundreds of infants and children. He is a member of NORD's Medical Advisory Committee. More.

View the video.

Guttmacher Named Director of NICHD

Alan Guttmacher, MD, has been named director of the Eunice Kennedy Shriver National Institute for Child Health and Human Development (NICHD) at the National Institutes of Health. Dr. Guttmacher, a pediatrician, is well known to the rare-disease community, and NORD applauds his appointment. Read the NIH press release.

Amazing Grace: Paula Shal Learns to Live with Rare Disease

When Paula Shal was growing up in a tiny town in Illinois, she was the smallest child in her class, and the sun bothered her eyes so that her older brother sometimes had to lead her to school. Later, Paula found she had a disease so rare most people have never heard of it. More.

Dorman Testifies Before Senate HELP Committee

NORD Vice President for Public Policy Diane Dorman presented invited testimony today before the Senate HELP Committee on promoting the development of treatments for rare pediatric diseases. Dorman talked about the need for pre-clinical research tools, federal funding for research, and reduced regulatory uncertainty. She also called for a greater emphasis on rare diseases in the nation's medical schools and for help in resolving issues that keep patients from accessing treatments. Read her testimony. Read NORD's press release.

NORD's Diane Dorman with Senator Sherrod Brown, NORD advocacy intern Jason Barron, and Jason's sister, Megan, who has epidermolysis bullosa, at the Senate HELP Committee hearing.

View Webcast of Senate HELP Committee Hearing

NORD's Diane Dorman and others are presenting testimony related to developing treatments for rare and neglected pediatric treatments. View Webcast.

NORD Reports Appropriations Progress

NORD has been notified that funding increases are being proposed for 2011 in two key areas in which NORD has been strongly involved in advocacy. Read the email sent to NORD members.

NORD Continues Support for GINA Regulations

NORD has signed on to a letter to the U.S. Equal Employment Opportunity Commission (EEOC) to express continued support for the Genetic Information Nondiscrimination Act (GINA). The EEOC is in the process of developing regulations for Title II of GINA, the segment of the law that ensures that employers can only obtain or request genetic information from employees on a voluntary basis.

Many companies conduct health risk assessments (HRA) for employees that include questions about family history and genetic information and sometimes offer monetary rewards or other gifts for completing the HRA. NORD advocates that no exemption to Title II of GINA should be made to permit requiring this information and potentially withholding rewards if genetic information is not divulged. Read the letter.

NORD Announces Rare and Neglected Diseases Caucus

U.S. Representatives Joseph Crowley (D-NY) and Fred Upton (R-MI) have formally announced the newly-established Rare and Neglected Diseases Caucus. As co-chairs of the Caucus, Crowley and Upton will focus on bringing Congressional attention to the 6,800 known rare diseases that currently have no approved therapies; ensuring sufficient funding for research and orphan product development; exploring ways to incentivize companies to create new drugs, biologics and humanitarian use devices; and providing an opportunity for Members of Congress, families and advocacy groups to exchange ideas and policy concerns.

Please encourage your Representative to become a member of this important Caucus. More information.

NORD Board Chair Presents Opening Testimony at FDA Hearing

Frank Sasinowski, chairman of the NORD Board of Directors, was the first speaker this morning in a two-day public hearing on the Food and Drug Administration's policies and procedures related to rare diseases and orphan products. Noting that only about 200 of the nearly 7,000 rare diseases have FDA-approved treatments, Sasinowski spoke about the need for flexibility and reduced regulatory uncertainty. Read the statement.

View slides submitted to FDA at the hearing by NORD's Vice President of Public Policy, Diane Dorman.

Text of opening remarks made by Timothy Cote, MD, Director of the FDA Office of Orphan Products Development.

Dorman Presents Testimony at Subcommittee Hearing

Diane Dorman, NORD's Vice President for Public Policy, presented testimony at a June 23 hearing on FDA's review process for rare and neglected diseases before the Senate Committee on Appropriations Subcommittee on Agriculture, Food and Drug Administration, Rural Development and Related Agencies. Read her testimony.

FDA's Chief Scientist and Deputy Commissioner for Science and Public Health, Jesse L. Goodman, MD, MPH, also presented a statement at the Subcommittee hearing. Read his remarks describing FDA's Efforts on Rare and Neglected Diseases.

Basketball Legend's Death Shines Spotlight on Little-Known Syndrome

The recent death of humanitarian and basketball superstar Manute Bol has focused attention on a rare but very serious syndrome that can occur as a result of taking many medications, including over-the-counter drugs. The Stevens Johnson Syndrome Foundation, a NORD Member Organization, has issued a press release to clarify information being reported about the syndrome. Read the press release.

NORD and 16 Others File Amicus Brief to Protect Health Care Reform

Arguing that the health care reform legislation is a package of separate but related parts, all of which are essential for ultimate success, NORD and 16 other patient advocacy groups have filed an amicus brief in the U.S. District Court for Eastern Virginia. Their concern is that the Virginia Attorney General's efforts to overturn one part of the health reform package will jeopardize insurance reforms such as the elimination of pre-existing condition clauses. Read NORD's press release.

View Slides from NORD Corporate Council Meeting

NORD's Corporate Council met in Washington DC on May 18. Topics included the NIH/FDA interface, healthcare reform and rare diseases, and accelerating orphan product development. View slides.

Rare Disease Innovators Honored at NORD Gala

More than 500 people from all segments of the rare disease community attended the annual NORD Gala on May 18. Shown here are NORD President and CEO Peter L. Saltonstall, guest emcee Patricia Richardson, EURORDIS CEO Yann Le Cam, and NORD Board Chair Frank Sasinowski.

View Washington Life magazine photos of NORD Gala

NORD Issues RFPs for 2010

As a result of donations from individuals and patient groups, NORD has posted six new Requests for Proposals (RFPs) for clinical studies of five rare disorders: APS Type 1, Homocystinuria, Larsen Syndrome, Pseudomyxoma Peritonei, and Stiff Person Syndrome. To read NORD's RFPs and/or to download the applications, go to http://www.rarediseases.org/research/requests.

Charity Navigator Awards 4-Star Rating to NORD

NORD has been given a top (4-star) rating by a leading independent evaluator of nonprofit organizations, Charity Navigator. The 4-star rating honors charities that "consistently exceed standards and outperform most others" in effective use of donated funds. Details.

Social Security Announces 38 Additional Compassionate Allowance Conditions

The Social Security Administration (SSA) is adding 38 more conditions to its list of Compassionate Allowances. These are severely disabling conditions that qualify for expedited review for disability assistance. View the SSA press release and list. The conditions were identified following public hearings and with input provided by medical experts from the National Institutes of Health, NORD's Medical Advisory Committee, and others.

PNH Meeting to Take Place August 14 in Nashville

NORD is hosting a series of regional support meetings for patients and families affected by paroxysmal nocturnal hemoglobinuria (PNH). Details for Saturday, August 14 in Nashville. For more information please contact solivo@rarediseases.org.

Future meetings to be held in Ann Arbor on September 14th and Austin September 18th. Please check back to see future meeting dates and locations.

Past PNH Presentations:

Dr. Martin Weltz - Baltimore

Dr.Gabrielle Meyers - Portland

Dr Charles Farber - Boston

Dr. Mary Ann Skiba - Denver

Dr. Jamile Shammo - Chicago

Dr Ilene C Weitz - Los Angeles

What is PNH? Power Point presentation download.
If you do not have Power Point on your computer, you can download a free viewer here.