Recognizing the enormity of the challenges our BPAN community faces, BPAN Warriors is dedicated to making a lasting impact through our campaigns and programs. While our efforts are driven by our organization’s singular focus, we spread a wide net by investing in a variety of progressive strategies. Learn more about our initiatives and get involved yourself.
BASIC SCIENCE: MOUSE MODELS, PATHWAYS AND CELL LINES
Rare disease research is often hampered by:
Biospecimens with poor correlative information
Improperly stratified experimental cohorts
Small sample sizes
BPAN Warriors recognizes that to accelerate research we must provide researchers the "basic" tools to perform required laboratory studies. Mouse models, cell lines (iPSC) and a firm understanding of potential targets for treatment are critical to expedite a treatment path.
DRUG REPURPOSING AND GENE THERAPY
ISOFORM | ALTERNATIVE SPLICING ANALYSIS
The goal of the isoform analysis is to evaluate known BPAN isoforms and identify/validate which of the isoforms may be the best candidate to deliver via AAV gene therapy. Because multiple WDR45 isoforms exist, we will need to target the isoform(s) with the highest protein expression or those that have the highest impact in neural/cellular function.
FAU G-CSF GENE THERAPY | PROTEIN THERAPY
A ground-breaking approach to treating BPAN. While it is not the "cure", G-CSF Gene Therapy has the potential to provide neuroprotection, stimulate cell growth of new neurons, improve mitochondrial function, reduce endoplasmic reticulum stress, and provide neuronal homeostasis. It has also shown improvement in cognitive function and motor skills in Parkinson's, Alzheimer’s and ischemic stroke mouse models.
COLLABORATIVE DATA SHARING
We see every challenge as an opportunity, and this initiative helps us ensure that our partners are better equipped with patient data and research findings, shared through our BPAN RACE platform. We are invested in an innovative approach that empowers our community and delivers the results needed to advance research.
Because many of the many research projects that occur globally, BPAN Warriors will provide a structure and guidelines to allow multiple collaborators, to work collectively throughout research/study execution, while providing assistance with project management and scientific advice.
Through collaboration we hope to:
• Foster connection between multiple researchers
• Promote cooperation
• Improve outcomes
• Increase the speed of project completion
BPAN RESEARCH AND COLLABORATIVE EXCHANGE (RACE)
CONNECTING OUR EXPANDING GLOBAL COMMUNITY
BPAN Warriors is 100 percent committed to accelerating treatments and a cure for BPAN. The symptoms of BPAN have been under-addressed in the research community for far too long. Research aimed at identifying treatments for the motor dysfunction, seizures and behavioral characteristics of BPAN is one of our top priorities.
A cure for BPAN is within reach but will require the expertise and collaboration of the best and brightest minds from a variety of fields in science and research.
A true research collaboration, and the infrastructure required to advance a cure for BPAN, is nonexistent. The BPAN Research and Collaborative Exchange addresses this challenge by creating the first-of-its-kind BPAN RACE Initiative, placing patient priorities squarely at the front of all research.
BPAN RACE is a patient-centered initiative, which supports and demands collaboration to identify, characterize and implement new therapeutics for the treatment and ultimate cure of BPAN. We require collaboration, transparency and ongoing communication with those who are most affected - our patient community.
With limited resources, and expanding global community, establishing tools for data sharing and research collaboration is imperative to expedite research.
PUBLICATIONS AND RELATED SCIENCE
Our BPAN Warriors community wants nothing more than to see patients with BPAN identified accurately and expeditiously, with the hopes that this may alleviate the emotional, physical and often financial burden that many families experience. To this end, we hope that the scientific and medical community will join us as we as we advocate and work to increase BPAN awareness throughout the world.
Provides immediate insight into the published genomic research for every disease and DNA mutation found in the medical literature. Genetic evidence is readily accessible through a search interface, where results are prioritized to highlight the most clinically relevant information. Mastermind is used by hundreds of genetic labs around the world to assure comprehensive genomic interpretation, and by pharmaceutical companies to understand the genomics associated with any disease.To view the list of variants tied to BPAN and request access to the genomic landscape, go to:
PUBMED US NATIONAL LIBRARY OF MEDICINE NATIONAL INSTITUTE OF HEALTH
PubMed comprises more than 30 million citations for biomedical literature from MEDLINE, life science journals, and online books. Citations may include links to full-text content from PubMed Central and publisher web sites.
Search for genes or proteins and their functional terms extracted and organized from over a hundred publicly available resources.
HELP US ACCELERATE RESEARCH
Get Involved Today
BPAN CENTRALIZED BIOREPOSITORY AT THE CORIELL INSTITUTE
BREAKING DOWN BARRIERS TO RESEARCH PROGRESS
We see every challenge as an opportunity, and this initiative helps us ensure that our research and scientific partners have the necessary tools to perform essential studies on patient derived samples. Making patient samples available to the entire research community in one centralized location reduces time and patient burden.
BPAN R.A.C.E. BIO-REPOSITORY
Blood, skin, biopsy, saliva and microbiome kits
Custom kitting and microbiome kits
Sample collection, informed consent and IRB
Public, private and independent biobanks
Sample collection, processing, cryopreservation and safe storage
Repository information management systems
BPAN NATURAL HISTORY AND PATIENT DATA COLLECTION PLATFORMS
BPAN PATIENT DATA ESSENTIAL FOR DRUG DISCOVERY AND PATIENT CLINICAL CARE GUIDELINES
The NBIA Research Group at OHSU has developed a new study called BPANready. The purpose of this study is to help us better understand the natural history of BPAN, meaning how symptoms appear and change over time. By studying individuals with BPAN, we also hope to identify disease markers that can be used in future clinical trials. A disease marker is any symptom or measurement that happens reliably in a disease, changes predictably with disease progression, and becomes “better” with successful treatment. A disease marker could be an MRI finding, a protein level in the blood, or a rating scale to measure symptoms or function. Natural history studies provide data that serve as the foundation for future drug trials.
BPAN RACE GRANTS
APPLY FOR A GRANT
BPAN RACE is committed to bringing practical treatments for BPAN into current medical practice as quickly as possible. While our priority is on funding translational and clinical research, we also support high-risk/high-reward discovery projects. BPAN RACE’s research program awards grants to the world’s best scientists who are focused on discovering and developing treatments, technologies and ultimately a cure benefiting those with BPAN. The focus of BPAN RACE-funded research is in supporting projects that have the potential for immediate and high impact on the BPAN community; therefore, priority will be given to research-based translational grants or those showing high promise for translating basic biomedical knowledge into clinical application.
BPAN RACE’s research program funds are raised exclusively through donations and grassroots fundraising activities initiated by BPAN RACE, our supporters, and families of children with BPAN. We have a duty to ensure that this money is used to fund the highest quality research that has the greatest chance of having an impact on the lives of children and families affected by BPAN. Because BPAN research falls into one of the most rapidly advancing fields of science (autophagy), BPAN RACE accepts innovative grant proposals on a rolling basis.
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