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Collaborations will lead to 5 NBIA grant awards in early 2019

September 2018

The NBIA Disorders Association is collaborating on five grants that will be awarded early next year, three of which are dedicated to studying BPAN, the fastest-growing NBIA diagnosis. The other two disorders eligible for grants are MPAN and PKAN.

Researchers have been notified to submit applications for the grants this fall, and representatives of our organization will be involved in the award-selection process.

Our first call for applications is for two grants of $51,020 each to study BPAN, or Beta-propeller Protein-Associated Neurodegeneration. Those applications involve a two-step process: submitting a letter of intent, undergoing a review and being asked to submit a full application, due Oct. 15.

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NBIA organization awards two grants for BPAN, including the first for an early-career researcher

September 2018

Seeking to attract more scientists to study NBIA, the NBIA Disorders Association this spring awarded it’s first-ever early-career grant to a researcher. That scientist will receive a total of $150,000 spread over two years to study BPAN.

 In addition, the organization awarded a one-year $45,000 grant to another BPAN researcher.

The recipient of the early career award is Dr. Young-Ah Seo, an assistant professor of nutritional biochemistry in the department of nutritional sciences at the University of Michigan School of Public Health in Ann Arbor. The NBIA Disorders Association board created this grant category to support highly promising early-career investigators as they transition from training to independence. The board hopes recipients will maintain an interested in the disorders and contribute substantially to NBIA discoveries throughout their careers.

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Deadline looming to seek BPAN research grants

May 2018

Hurry! June 15 is the deadline to apply for NBIA Disorders Association research grants to study Beta-propeller Protein-Associated Neurodegeneration (BPAN).

The association plans to award two grants for BPAN research with money raised exclusively by BPAN families.

One is a $45,000 grant that is open to all scientists. The organization is interested in projects that have the potential to generate essential resources for the scientific community, advance knowledge about NBIA disease processes and produce preliminary data so that additional national and international funding can carry the work forward.

The other grant is for an early-career faculty investigator grant — our first such offering. Applicants must be within five years of their first faculty appointment, or the equivalent. The selected recipient would get up to $75,000 each year for two years, with an option for a third year, depending on progress.

This grant is intended to support highly promising but relatively new researchers as they transition from training to independence. The goal is to engage an investigator who will contribute substantially to this field for the duration of his or her career.

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Scientists report on progress of current NBIA research projects

May 2018

One of the key activities of the NBIA Disorders Association is awarding research grants, most of it with money raised by our hardworking families.

The board of trustees receives invaluable help from our Scientific and Medical Advisory Board, which helps set research goals, evaluates proposals and monitors projects after the board funds them. In evaluating proposals, the advisory board puts greatest priority on new paths of study that could lead to a treatment or a cure.

Throughout the process, the advisory board follows high ethical standards. For example, researcher-advisers who submit a proposal or have a conflict of interest must state their conflict and recuse themselves from decision-making. After the board of trustees approve a grant, it holds grantees accountable for meeting deadlines, delivering the promised work and providing updates to share with families. These recipients must submit regular financial and scientific reports to our SMAB; payments are made in stages after those reports are submitted.

Since 2002, the trustees have funded 33 research grants totaling $1,290,914. The board also has funded research contracts totaling $357,408, Hayflick lab funding at $250,000 in 2009 (when it was in jeopardy of closing) and part of a clinical consensus treatment guide at $16,117. All told, that’s nearly $2 million for research.

Here are updates on recent grants the board awarded:

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Nearly $150,000 in grants awarded for BPAN research

May 2018

Thanks to the fundraising efforts of BPAN families, research into this NBIA disorder is taking a leap forward this year with the award of three grants totaling $146,014.

Two of the grants went to Dr. Penelope Hogarth of the Oregon Health & Science University in Portland for a natural history study of Beta-propeller Protein-Associated Neurodegeneration, or BPAN. Our organization awarded her $45,000, and another $50,507 came from the University of Pennsylvania as a matching grant in collaboration with our organization.

Although an increasing number of BPAN individuals are being diagnosed, there is much to learn about how the disease progresses.

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Hayflick, Hogarth and Sibon team moving ahead on one of two drugs for PKAN

November, 2017

Drs. Susan Hayflick, Penny Hogarth and Ody Sibon told PKAN families recently that they are working with two companies to create a PKAN drug to ensure sufficient amounts for a clinical trial.

Speaking with PKAN families via a Facebook live stream video Nov. 6, the researchers said they are hopeful the compound that they are calling CoA-Z will correct a metabolic process involved in producing coenzyme A, called CoA. CoA is involved in metabolism and is thought to be low in individuals with Pantothenate Kinase-Associated Neurodegeneration, the most common form of NBIA. In the PKAN mouse, CoA-Z does everything Hayflick, Hogarth and Sibon would want to see before moving their studies into a human clinical trial.

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Board awards its first grants for FAHN research

August, 2017

The NBIA Disorders Association board in June awarded its first grants for two projects to research Fatty Acid Hydroxylase-associated Neurodegeneration, or FAHN, one of the NBIA disorders.

The grants were made possible by the fundraising efforts of the Engblom family from East Islip, N.Y. Parents Trevor and Gina, along with their son Kyle who has FAHN, worked tirelessly for over a year to raise the money.

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First patients in Retrophin trial to receive drug targeting PKAN


October, 2017

In July, the first patients taking part in an international clinical trial on a possible treatment for PKAN, the most common NBIA disorder, received Retrophin Inc.’s drug, fosmetpantotenate, also known as RE-024.

This long-awaited launch of Phase 3 of the trial, which Retrophin delayed until manufacturing issues were resolved, will assess the safety and effectiveness of RE-024. If the San Diego-based company is successful, RE-024 would be the first medication targeting the underlying cause of PKAN, or Pantothenate Kinase-Associated Neurodegeneration. It could change the course of the disease.

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Hayflick team announces work on 2 promising compounds for PKAN

August, 2017

Calling it “a big deal,” Dr. Susan Hayflick, who has been studying the NBIA disorders since the early 1990s, announced at the June family conference that her lab is working on two potential treatments for PKAN, the most common form of NBIA.

One is a previously approved U.S. Food and Drug Administration drug, which Hayflick didn’t name but said her lab had just begun testing in PKAN-impaired mice. It’s “pretty safe and inexpensive and available worldwide, but we have to see if it helps the mice” said Hayflick, a physician and researcher at the Oregon Health & Science University in Portland.

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Million Dollar Bike Ride nets $101,014 for BPAN research

August, 2017

A cycling team representing the NBIA Disorders Association raised over $50,000 for BPAN research and will have the full amount matched for taking part in the University of Pennsylvania Health System’s fourth annual Million Dollar Bike Ride for rare disorders.

Penn Medicine is now requesting letters of interest by Sept. 18, 2017, from the international scientific community for grants to study the diseases designated by the riders at the May bike ride in Philadelphia. Full applications are accepted by invitation only after letters of interest are reviewed.

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Retrophin recruiting for PKAN drug study

Retrophin logo


May 4, 2017

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 Inc. has begun to recruit patients for its planned clinical trial for PKAN patients.

The company plans to test a drug, fosmetpantotenate, the new name for RE-O24, to see if it can help patients with the most common NBIA disorder, PKAN. Retrophin had hoped to begin the study late last year, but a manufacturing problem caused a delay until now.

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Retrophin announces delay in starting RE-024 trial

Retrophin logo


JANUARY, 2017

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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