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Retrophin announces delay in starting RE-024 trial

Retrophin logo

The NBIA Disorders Association posts the following announcement for informational purposes only. While the organization supports and encourages the discovery of treatments for NBIA individuals and willingly posts information concerning research studies (such as questionnaires and clinical trial enrollment), we do not endorse specific studies. Nor do we advise NBIA individuals or their families to take part in a particular study. Rather, we believe that those decisions are best made by affected individuals and/or their families, in collaboration with their doctors.

Retrophin recently informed the NBIA Disorders Association that the phase three trial of its PKAN drug, RE-024, is being delayed because of a manufacturing problem.

The trial’s placebo - often called a sugar pill because some patients will get it rather than the drug -“did not meet the stringent quality standards necessary for a clinical trial,” said Tricia Sterling, executive director of patient care at Retrophin.

The company had planned to start dosing patients by end of 2016 but now must correct the manufacturing issue so that the trial can proceed safely and generate high-quality clinical data, she said.

Delays in starting clinical trials are common for a variety of reasons.

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Next round of NBIA research grants to focus on BPAN and FAHN

- December, 2016

Families who raised money for studies on two NBIA disorders, BPAN and FAHN, will soon see a focus on the diseases when the NBIA Disorders Association solicits a new round of study proposals.

Within the next few months, the association expects to specifically ask for grant applications that address research priorities for BPAN and FAHN.

The association’s Scientific & Medical Advisory Board is in the process of setting research priorities for Beta-propeller Protein-Associated Neurodegeneration (BPAN) and Fatty-Acid Hydroxylase-associated Neurodegeneration (FAHN). Those priorities will be used to guide research proposal requests, and the grants will be awarded as soon as possible in 2017.

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Identifying NBIA research priorities key topic at meeting of families, researchers and board

- August, 2016

As the NBIA community grows with more disorders under its umbrella, the organization’s research agenda also is evolving as some families seek more attention—and dollars—for their loved one’s disorder.

That was among the topics discussed by families, the NBIA Disorders Association board and its Scientific & Medical Advisory Board at a meeting on research priorities. It was held during the association’s 20th anniversary celebration in the Cincinnati area.

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Two more researchers join association’s Scientific & Medical Advisory Board

- July, 2016

A group of researchers, physicians and technology executives who provide scientific and medical advice to the NBIA Disorders Association has added two new members, bringing the total to seven.

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Researchers in Portland host first-ever meeting with BPAN families

- June, 2016. By Matt Dyer and Meg Talley Dyer

For the first time, researchers at the Oregon Health and Science University hosted a meeting in Portland for BPAN families so they could share information, take biological samples and introduce families whose loved ones share the same disorder.

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Organization wins award from DonorPro

Donor Pro logo

The NBIA Disorders Association is pleased to announce that it has won an award from DonorPro, a donor management and fundraising software provider.

Our organization partners with DonorPro to help us raise more money and efficiently manage our relationships with donors. We won the award for "Best Digital Fundraising Performance." We received a check for $250, a certificate and valuable consulting services and training. 

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Request for Board & Development Members

Board of Trustees

September 5, 2015

Dear NBIA Community,

Do you have a skill, an area of expertise or a passion to help improve the lives of NBIA individuals and their loved ones? If so, the NBIA Disorders Association Board of Trustees wants you!

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New research policy taking effect

Research Lab

The NBIA Disorders Association Board of Trustees recently approved changes to the organization’s policy on research donations to add flexibility and to acknowledge the importance of donors being able to designate their gifts to specific disorders. Here’s a summary of the policy:

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NBIA Disorders Association board awards nearly $160,000 in grants

The NBIA Disorders Association board chose three projects among 19 applications to receive grants totaling $157,725 this year, enabling researchers to study three different forms of NBIA.

The grants will further research into Pantothenate Kinase-Associated Neurodegeneration, or PKAN, Mitochondrial-membrane Protein-Associated Neurodegeneration, known as MPAN, and Beta-propeller Protein-Associated Neurodegeneration, or BPAN.

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Meet Drew


Drew is a 20 year old with NBIA disorder. He is passionate about wanting people to know about rare diseases. Watch and share this inspiring video of his story.

Watch Video

2015 Research Grant Cycle Open for Applications


Researchers interested in applying for a grant from NBIA Disorders Association have until April 1, 2015 to submit their application.

Grants will be for one year and up to $45,000 with funding to begin June 30, 2015. Proposals to develop models will be considered for multi-year funding with budget justification.

Please see our grant application for more details:
Grants Program Information

OHSU Awarded Research Grant for 2014 PKAN Grant Round

Dr. Susan Hayflick

Dr. Susan Hayflick of Oregon Health & Science University has been awarded $45,000 by our organization for her grant titled, “Examining the role of ischemia in the neurodegenerative disease PKAN.” The one year grant period began on December 15, 2014.

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NBIAready Project Launches Website

OHSU group

The OHSU team has a new website at www.NBIAcure.org They created the site to serve as a valuable resource that is easy to use and understand.  It is part of a new project called NBIAready. It has three components: long-term studies of individuals with NBIA, development of a powerful database and the development of this comprehensive website to aid families, clinicians and researchers.

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Eighth International Family Conference May 28-31, 2015

Eighth International Family Conference May 28-31, 2015

Our Eighth International NBIA Disorders Association Family Conference will be held May 28-31, 2015.

Site of the conference is the Embassy Suites Minneapolis North in Brooklyn Center, Minn. The hotel is approximately 7 miles from downtown Minneapolis. Room rates are $114 for up to 2 people and $10 for each additional person.

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Board awards 3 research grants in March totaling $239,680 to study NBIA

Research Grant

The NBIA Disorders Association board is awarding $239,680 to three research projects that received top scores from a pool of 22 grant applications.

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NBIA research advances with grant awards

Dr. Penny Hogarth

NBIA researcher Dr. Penny Hogarth has been awarded startup funds to pursue a new area of NBIA study and to develop tests for gauging severity of the illness.

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Focus on Issues Radio

Patricia Wood

President Patricia Wood was the guest speaker on a “Focus on Issues” segment at KECR 910 AM radio station in El Cajon, CA in December.


SCOPE Summit

SCOPE Summit

Miami, February 4-6, 2014

President Patricia Wood will participate in a panel discussion on "Partnership with Patient Communities to Get from Data to Clinic” at the Cambridge Healthtech Institute's SCOPE Summit in Miami.

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New Research Grant cycle open for applications


Researchers interested in applying for a grant from NBIA Disorders Association have until October 31 to submit a letter of intent.

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Two clinical trials underway for PKAN

Clinical Trial

Listed in this news article are two clinical trials that may be of interest to you. Click the button below for more information about them.

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2013 Family Conference

The Alamo

Record number attend San Antonio gathering and enjoy learning and sightseeing together

Our Seventh International Family Conference in San Antonio was our largest yet, with 120 participants from the U.S., Canada, Australia, Norway, England, India and Japan. That number included 27 NBIA individuals, also a record number.

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New Google Group Forum for NBIAdisorders

NBIA Disorders Google Group

Our listserv through the Genetic Alliance is no longer available and we have moved to Google Groups.

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