In a pandemic they talk about the importance of time. Delay the spread. Flatten the curve. Give us time and we'll have the infrastructure, processes and people in place to give us the best chance of avoiding the worst case scenario.
Time is vital.
In our community of Sanfilippo families and researchers, time is also vital, but it's different.
Every day that we don't have a treatment for Sanfilippo is a day this disease progresses and children decline. We don't want to delay the research, pause the progress, defer the hope.
Your big-hearted and generous support of Sanfilippo research continues to amaze us. And with your help we've committed $4.7 million to 24 projects in the search for a cure for Sanfilippo.
During this unprecedented time, we want to update you on how the COVID-19 situation is impacting children and their families and the critical Sanfilippo research already underway.
The vulnerability and resilience of Sanfilippo families
- Children with Sanfilippo and their families are among some of the most vulnerable people in our communities. Lauded already for their incredible resilience in the face of this disease, the added challenges of COVID-19 make life substantially more difficult
- The isolation, the demand on carers, clinicians and disability support services, and the heightened risk to the health of these children with such a devastating pre-existing condition, all add to the uncertainty and anxiety these families are facing
- Specifically, families feel higher anxiety due to the risk of upper respiratory tract infections associated with COVID-19. Respiratory problems are one of the leading causes of death of children with Sanfilippo.
Research is as urgent as ever in the search for treatment
- Research to find treatments for children with Sanfilippo is no less urgent in the current pandemic. Sources of significant research funding, such as Government and Major Philanthropy, along with research personnel including clinical staff, are being diverted towards COVID-19 research and support, reducing the funding and personnel available to progress research for Sanfilippo
- Clinical trials testing potential Sanfilippo treatments are navigating a difficult balancing act: keeping vulnerable children safe from exposure to COVID-19 while attempting to continue these vital trials. Some trials have been able to adapt using telehealth and postponing assessments, but sadly recruiting new participants will be delayed for all trials
- While some research labs overseas have been closed, putting a pause on research progress, Sanfilippo researchers are applying for funding in record numbers so that they can hit the ground running as soon as labs are open again
- Fortunately key projects, particularly in Australia, such as our ‘Brain in a Dish’ project are able to proceed and our researchers are working hard to make strides forward for Sanfilippo
- Our research project pipeline is maturing. Projects that started in 2017 are now moving potential treatments closer to the clinic and consequently more expensive drug development stages, which increases our funding requirements
Our commitment has never been stronger to drive research for a world without Sanfilippo Syndrome. Your support has never been more important.
Setting up a regular monthly gift provides ongoing funding for research into treatments for children with Sanfilippo.
We hope you remain safe and well during this time.
