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  • NBIA Alliance News

Hayflick team announces work on 2 promising compounds for PKAN

Dr. Susan HayflickCalling 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. ...More


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

Million Dollar Bike Ride logo

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. ...More


Retrophin recruiting for PKAN drug study

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 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.

In the clinical trial, which takes its name from the drug and is being called the FORT Study, Retrophin plans to recruit 82 people diagnosed with PKAN in North America and Europe. Eligible patients must range in age from 6 to 65, be able to breathe without a ventilator, must be off of deferiprone for 30 days and cannot have had a deep brain stimulation device implanted within the past six months.

For information about the study, trial sites and screening requirements for participants, visit www.pkanfortstudy.com. Information about the study is also available on www.clinicaltrials.gov.


6th International Symposium on NBIA & Related Disorders

Meeting logo

April 7-8, 2017 - Skamania Lodge, Stevenson, Washington, USA.

ATTENTION RESEARCHERS: Researchers studying NBIA and similar neurological disorders will be sharing the latest advances in their work at this symposium.   ...More


9th International NBIA Disorders Association Family Conference

Hyatt Deerfield exterior frontJune 1-4 - Deerfield, IL

The NBIA Disorders Association’s Ninth International Family Conference is set for June 1 to 4 in Deerfield, IL, just 30 minutes north of Chicago and its many attractions: Millennium Park, the famed Field Museum of Natural History and the exceptional Art Institute of Chicago.  ...More


European grant will help researchers test drugs and supplements for MPAN


With a grant from our sister organizations in Switzerland and the Netherlands, researchers in Germany are testing potential treatments for MPAN, a form of NBIA.

The scientists plan to use the grant of 50,000 euros from NBIA Suisse and Stichting Ijzjersterkthe to test about 200 drugs and nutritional supplements that might help MPAN patients. They will screen those compounds in a fruit fly model of MPAN.  ...More


Italian lab receives grant, drug access in quest to uncover PKAN treatments

italian alliance news1A lab in Italy working on the role of coenzyme A in NBIA received a grant of 16,500 euros in June from the Italian Association for Neurodegeneration with Brain Iron Accumulation (AISNAF).

The funding, which could be renewed at the same amount, will enable the lab to continue its work on CoA for six months while waiting for larger grants to come in.  ...More


NBIA Suisse joins NBIA Alliance

nbia suisse logoThe NBIA Alliance, an informal umbrella organization for the lay advocacy groups, recently welcomed its newest member, NBIA Suisse from Switzerland. Set up in 2012, the alliance now has eight member organizations. In addition to the Swiss and U.S. groups, they are in Canada, France, Germany, Italy, the Netherlands and Spain.

The newest alliance member, NBIA Suisse, came on board in October, 2015. Fatemeh Mollet is the founder and has three nieces with a form of NBIA known as Mitochondrial-membrane Protein-Associated Neurodegeneration, or MPAN.  ...More


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