This short video provides valuable information about the NBIA Disorders Association network of support services for newly diagnosed NBIA families.
Families and Researchers Share, Learn and Support those dealing with ultra-rare NBIA Disorders
Deerfield, IL NBIA Disorders Association, a non-profit organization that provides support to families, educates the public and accelerates research with collaborators from around the world, today announced that it will hold its Ninth International Family Conference on June 1 to 4 in Deerfield, IL. With over 200 attendees from around the world coming together to support one another and learn about research to find cures and treatments, this is the largest gathering of families and researchers ever held for this group of ultra-rare diseases.
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.
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. ...More
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. ...More
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
June 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
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
A 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
The 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