This short video provides valuable information about the NBIA Disorders Association network of support services for newly diagnosed NBIA families.
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. ...More
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
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. ...More
On February 28, 2017, the tenth edition of Rare Disease Day will see thousands of people from all over the world come together to raise awareness for rare diseases. This year’s theme is “Research.” ...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