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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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