GLOBAL RARE DISEASE PARTNERS

EMPOWERING THE PATIENT COMMUNITY

WORKING TOGETHER WITH A SHARED MISSION

While BPAN continues to be an ultra-rare disease, with a population that is widely scattered globally, BPAN Warriors and multiple patient advocacy organizations exist today that share a common mission: to improve the health and welfare of individuals who live with BPAN. Part of this mission is to identify the most promising research that may lead to treatment opportunities that will stop the progression of this devastating disease, provide reliable symptom management and ameliorate daily healthcare challenges, advocate for fair and equitable access to healthcare, homecare, equipment and diagnostic services, and ultimately, find a cure for BPAN.

 

Our collective experience and shared knowledge is the cornerstone of our global mission. We continue to work with our global partners to ensure that we work together towards our common mission. Together we strive to provide BPAN specific resources, research updates and most importantly facilitate ongoing  connection between our BPAN patient families.

 

While there is work to be done, we are proud to be a part of a global network of collaborative partners who understand the importance of patient-centered initiatives and realize how critically important it is to place the patient front and center.

BPAN AND NBIA PATIENT ADVOCACY ORGANIZATIONS

RARE DISEASE PARTNERSHIPS

"The average length of accurate diagnosis of a rare disease is 4.8 years and involves more than seven physicians or specialists who may be geographically distributed. The genetic diagnosis may not be directly associated with any treatment options, and physicians will continue to treat symptoms, albeit in a more informed way based on likely prognosis of the case. Therefore, genetic diagnosis could be of benefit beyond treatment management as it can offer information to families, many who just want to know what is wrong with their family member and can also inform fertility decisions."

- Weida Tong, PhD

 

Rare Disease Day: A Day of Global Rare Disease Awareness

The last day of February each year promotes awareness of rare diseases and the patients that suffer from them, as well as access to medical treatment and representation. Advocacy is critical for rare disease patients and their families. Whether it is improving disease understanding, connecting families with one another and with researchers, fundraising for research, or sharing resources, rare disease initiatives provide much needed support to patient communities. The National Organization of Rare DisordersPerlara, the Rare Genomics Institute, the Iberoamerican Alliance for Rare Diseases, and Global Genes are just a few organizations that have provided meaningful outcomes for patients and families. Meanwhile, Orphanet and NIH – GARD (Genetic and Rare Diseases Information Center) gather and share information on thousands of rare diseases, along with other resources, to improve diagnosis, care, and treatment.

Access to worldwide promotion of this event can be found via #RareDiseaseDay.