A year it all started – reflecting on 2024 at Cure HSPB8
As we approach the close of 2024, we at Cure HSPB8 are filled with gratitude and pride for the progress made this year. Here are some of the highlights that shaped this year:
Cure HSPB8 became an official charitable project under the auspices of Social and Environmental Entrepreneurs on April 2, marking a significant milestone that formalized our mission. This achievement was further complemented by a transformative donation from the Volo Foundation in May, which has fuelled our initiatives and broadened our reach. Our team also grew significantly: Todd King and Dr Ania Kordala co-founded Cure HSPB8, while Dr Karolina Chwałek and Dr Matt McLeod joined us in April, Khosiyat Makhmudova in May, and Julia Mielcarz in June.
Research and Development
In January, we launched a global patient registry, a pivotal step in uniting patients, researchers, and clinicians to better understand HSPB8 Myopathy.
A major highlight is the start of our collaboration with Everlum Bio and XYZ Laboratory, working, amongst other things, towards developing induced pluripotent stem cells (iPSCs) and a humanized mouse model for HSPB8 Myopathy. This partnership began a detailed amenability study to explore therapeutic strategies. Additionally, we started working with Dr Wenli Zhou from XYZ Laboratory on an innovative project to measure autophagic flux using blood samples. This cutting-edge approach could pave the way for efficient evaluation of potential interventions, ranging from fasting protocols to pharmacological treatments.
September marked the launch of the Cure HSPB8 Research Meetings, creating a platform for collaboration and knowledge sharing among researchers and stakeholders. Earlier in the year, in March, we attended the MDA Conference in Orlando, where we presented a poster and gained valuable insights from other patient advocates. We also presented at another conference on ultrarare diseases, highlighting the crucial role of patient groups in drug development processes.
Awareness, advocacy, community
Our efforts to expand awareness and advocacy saw significant growth. Thanks to Karolina’s and Julia’s efforts, we established a presence on Twitter, Facebook, and LinkedIn, supported by a dynamic social media campaign. Our website also expanded significantly, complemented by an active newsletter, mailing list, and a Google Ads campaign. Joining the Global Genes Alliance further connected us with other rare disease organizations, amplifying our collective impact.
Moreover, HSPB8 Myopathy was officially added to the MDA website as a type of myofibrillar myopathy, enhancing visibility and recognition. Cure HSPB8 was also included in the Orphanet database, ensuring that individuals searching for information can easily find our organization.
In addition to these achievements, an advertisement featuring Cure HSPB8 was published in LGMD Magazine, raising awareness within the broader muscular dystrophy community. These efforts have helped us reach new audiences and strengthened our advocacy initiatives.
As we reflect on 2024, it has been a transformative year filled with milestones that strengthen our foundation and expand our potential to make a difference. Looking ahead to 2025, we are inspired to build on this momentum, advancing research, supporting our community, and advocating for a brighter future for those impacted by HSPB8 Myopathy.
Thank you for being an integral part of our journey. Together, we are making strides toward hope and healing.