English French German Italian Spanish
  • Photos & Videos from the 2017 Conference

    June 1-4, 2017 - Chicago, IL
    View Photos & Videos

  • Corporate Affiliate Program

    Can your corporation help us continue our work for our loved ones?
    More Information

  • The story of a newly diagnosed NBIA family

    This short video provides valuable information about the NBIA Disorders Association network of support services for newly diagnosed NBIA families.
    Watch Video

  • 1
  • 2
  • 3
  • News
  • Events
  • NBIA Alliance News

First patients in Retrophin trial to receive drug targeting PKAN

FortStudyLogo

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. ...More

 

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

 

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

researchers

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

 

Text Size

Dream Big Monthly Giving

Drew and his mother walking down a hallway

NBIA Alliance

Donate Now

Rare Connect

Connect with Us - NBIA Families and Friends

Sign-up for our Newsletter