News from research
Here you will find current information on NBIA research and related research areas.
Update on the CoA-Z clinical trial
Over the past several months, the OHSU CoA-Z study team has shared the initial study results with all CoA-Z study participants and their families, as well as the broader NBIA community of families, friends, clinical providers and supporters. It follows...
NBIA International Scientific Conference in Poland
On October 19 and 20, 2023, a BPAN workshop, an NBIA Alliance meeting and an international NBIA scientific conference were held in Warsaw, Poland. There, researchers, physicians and patient representatives from many countries came together to discuss the...
Insights into BPAN research at the family conference in the USA
BPAN families attending the NBIA Disorders Association (NBIADA) Family Conference in Houston, Texas, in May received updates on BPAN research from four researchers, beginning with Kira Anderson, a study coordinator with the NBIA Disorders Association (NBIADA)....
BPAN researcher develops stem cell model and awaits funding for planned drug screening
Dr. Paul Lockhart of the Murdoch Children's Research Institute in Melbourne, Australia, says that while his BPAN research has developed an important "brain cell" model using stem cells from affected BPAN individuals, his next step - the...
International NBIA Symposium held in Switzerland in October 2022
In 2020, the last NBIA scientists' meeting took place only online due to the pandemic. In October 2022, for the first time since 2017, numerous actors in NBIA research finally met in person again for 3 days for the 8th International NBIA Symposium in...
Care guidelines for PLAN/INAD are now in progress
Thanks to the support of four organisations funding the project, consensus guidelines for treatment and care in a form of NBIA known as PLAN or PLA2G6-associated guidelines are now available.
INAD gene therapy is one step closer
Work on a gene therapy for infantile neuroaxonal dystrophy, called INAD, got a big boost in October when a London-based biotech firm announced its intention to help improve the...
PKAN study with CoA-Z completed in the USA
Oregon Health & Science University in Portland (USA) conducted the first clinical trial for PKAN patients in the USA from 2019 to 2022 with a new drug called "CoA-Z" for the treatment of PKAN. This first part of the trial has now been...
Two MPAN project grants totaling $140,000 awarded
Hoffnungsbaum e.V. in conjunction with three sister organizations in Europe and the US, has awarded two MPAN grants to advance research priorities in the field of mitochondrial membrane protein-associated neurodegeneration (MPAN)...
October 12, 2022, online lecture: Understanding hereditary developmental disorders better - what you can learn from the natural course of the disease
Steffen Syrbe is a pediatric neurologist and Professor of Pediatric Epileptology at Heidelberg University Hospital. He has been working with children with neurodevelopmental disorders for over 15 years. During this time, he has developed a strong interest in genetic causes...