Dr. Brady to Receive
One of Nation's Top Awards

September 3, 2008 — Roscoe O. Brady, MD, a member of NORD's Medical Advisory Committee, has been named a recipient of the nation's highest honor for innovative achievement in science and technology. On September 29, President George Bush will present Dr. Brady with a National Medal of Technology and Innovation.

Read our news release.

Social Security Administration
Seeks Input on Rare Diseases

People with rare diseases who apply for Social Security disability assistance are often turned down at first because their diseases are not on the approved list. To help the Social Security Administration address this problem, NORD is asking physicians with expertise on various rare diseases to help establish criteria related to those diseases. Read about a survey for medical professionals.

PNH Support Meetings

August 29, 2008 (update) — NORD is hosting a series of regional support meetings for patients and families affected by paroxysmal nocturnal hemoglobinuria (PNH).

NORD's Southeast Regional PNH Support Meeting will be held in Atlanta, Georgia on Saturday, September 13th and will take place at the JW Marriott Hotel located in the popular area of Buckhead. Our special guest speaker will be Dr. Carlos DeCastro, Director of the PNH Clinic at Duke University Medical Center.

The Northeast Regional PNH Support Meeting will be held in New York, NY on Saturday, September 27th. The PNH Research and Support Foundation's Third Annual Walk for PNH is taking place September 28, 2008 in New York City. This event is the biggest annual U.S. fundraiser dedicated to raising money to help fight PNH disease by funding much-needed medical research and patient support programs.

What Is PNH? (Presentation by Dr. Wendell Rosse, Professor of Medicine, Emeritus, Duke University Medical Center)

FDA Approves First
Bone Marrow Stimulator

August 22, 2008 — The Food and Drug Administration (FDA) today approved romiplostim (Nplate), the first product that directly stimulates the bone marrow to produce needed platelets in patients with chronic immune thrombocytopenic purpura (ITP). Platelets are the blood components that help with clotting, and people with ITP have too few of them.

Survey Being Conducted in Argentina

A research project is being conducted in Argentina to determine problems experienced by people with rare diseases.  This project could potentially lead to public policy changes to address these problems.  NORD has been helping Moira Liljesthrom, president of Fundacion FOP (fibrodysplasia ossificans progressiva) get the word out about this survey. People who live in Argentina who are interested in learning more about this project, may visit www.fundacionfop.org.ar or write to info@fundacionfop.org.ar.

FDA Approves First Drug
For Huntington's Disease Chorea

August 15, 2008 — The Food and Drug Administration approved tetrabenazine (Xenazine) for the treatment of chorea in people with Huntington's disease. Chorea is the jerky, involuntary movement that occurs in people with this disease. This is the first drug approved in the U.S. for any symptom of Huntington's disease. The manufacturer is Prestwick Pharmaceuticals, Inc.

NORD Celebrates
25 Years of Progress

More than 500 people attended a recent gala at Washington's Union Station in honor of the 25th anniversary of NORD and of the Orphan Drug Act. As a result of the Orphan Drug Act, more than 320 new drugs have been brought to market for rare diseases.

         

         

View NORD's 25th Anniversary Gala video

President Ronald Reagan signed the Orphan Drug Act in 1983, bringing new hope to the 25 million Americans with rare diseases. Throughout 2008, we’ll be celebrating the 25th anniversary of this very successful legislation and of the founding of NORD.

No Fall Conference This Year
Because of the special activities related to NORD's 25th anniversary celebration, there will be no fall conference this year.� However, watch this space for announcement of a major event in 2009.

Read our news release.

West Nile Virus

Now that summer is in full swing, several states are reporting confirmed cases of West Nile Virus. The CDC (Centers for Disease Control and Prevention) lists tips on its website for reducing your risk for this disease. Read the report on West Nile Virus from NORD's Rare Disease Database.

CDC Lists Steps to Reduce West Nile Risk

NIH Launches Undiagnosed
Diseases Program

MAY 19, 2008—NORD participated in a conference call this morning hosted by the National Institutes of Health (NIH) to learn about the introduction of the new NIH Undiagnosed Diseases Program. The goals of this trans-NIH program are to provide answers to patients with mysterious conditions that have long eluded diagnosis and to advance medical knowledge about rare and common diseases. Patients must be referred to this program by a physician or other health care provider. All cases submitted for consideration will be reviewed by a multidisciplinary team of specialists and those selected for participation will be evaluated at the NIH Clinical Center in Bethesda, Maryland. There is no charge for participating in this clinical research program.

Individuals with undiagnosed conditions who are interested in this program should call the Clinical Center Patient Recruitment Call Center (1-866-444-8806). Additional Information is available from the NIH Office of Rare Diseases at http://rarediseases.info.nih.gov/undiagnosed.

New Program to Help Families
Affected by PKU

BioMarin Pharmaceutical, Inc., has awarded a grant to NORD to help families affected by phenylketonuria (PKU) purchase the specialized medical foods required for people with that rare metabolic disorder. More information.

President Signs "GINA"

President Bush has signed into law the Genetic Information Nondiscrimination Act of 2008 (GINA). The act protects Americans against discrimination based on their genetic information in matters related to employment and health insurance. It is hoped that this measure, which was debated in Congress for 13 years, will help people take full advantage of medical treatments that are increasingly personalized, without fear of discrimination.

One in a Million®

On April 21, 2008, Kristine Bostek ran the Boston Marathon for the first time in her life. She did it to raise money for research on Dubowitz syndrome, a rare disorder that affects her niece, Caitlin Granger. “Caitlin was at mile #20. It was a very emotional part of the race for me,” Kris said afterward. She raised more than $10,000 and donations continue to come in, in Caitlin's honor. Read the letter Kris wrote to her friends and family.

Read other One in a Million stories.

Read About NORD’s
Patient Assistance Programs

NORD administers various programs to help people obtain certain medications. These include medication assistance programs for uninsured patients, Medicare and insurance co-pay programs, early or expanded access to investigational products, travel and relocation assistance for clinical trial participants, and emergency or limited access to products in short supply. Click here to read about the patient assistance programs currently being administered by NORD.

NORD Gets 4-Star Rating
for Sound Fiscal Management

For the fifth consecutive year, NORD has been awarded a top (4-star) rating for sound fiscal management by Charity Navigator, a leading charity evaluator. The evaluation shows that NORD spent slightly over three percent of its revenues on fundraising and administration. All the rest went directly to programs and services. Details.

NORD supports National Family Caregivers Month.