The Lysosomal Disease Network (LDN), a consortium organized under the NIH Rare Diseases Clinical Research Network program, aims to improve the treatment of lysosomal diseases by learning more about each of these conditions by funding a variety of projects including 1-year pilot studies. LDN intends to fund 1-2 pilot projects, on an overlapping annual basis, that promote innovative research at the forefront of new therapies/technologies/advancements for lysosomal disease. Pilot studies must be clinical in nature (either through the use of human subjects or human specimens) and push forward the overarching themes of the LDN: advances in clinical trial readiness, newborn screening, long-term outcomes, and global reach.
Complete application information and dates can be found here.
Utilizing NIH funding, the Lysosomal Disease Network provides $50,000-$75,000 total costs (direct and indirect costs) to a post-doctoral level researcher with an interest in a career in lysosomal diseases. The Fellowship is for one year, coinciding with the NIH funding cycle (August 1st to July 31st). The LDN Fellowship can support currently-ongoing research projects, as well as new research projects. The projects are required to be of a clinical nature, working with human subjects or human tissues; they cannot be non-human medical research.
Complete fellowship application instructions and dates can be found here.
Dear Dr. Whitley and WORLDSymposium team,
“I wanted to share some lovely feedback with you all regarding the ability for families to attend the meeting virtually this year free of charge … though we’d all prefer to be together in person, this is such a great opportunity to learn how we can increase access for patients and families to the amazing science routinely shared at the WORLDSymposium. Thanks for all you do!”
(Cara O’Neill, Chief Science Officer Cure Sanfilippo Foundation)
What Patient/Families have to say:
- This is great and actually really helpful for international families to be able to listen in vs the normal cost of having to fly to the US. Hope they keep a virtual attendee option in future for patients and families at least.
- Amazing opportunity for international patients/families.
- … it’s not just the cost but also the practicality of leaving a child/ children behind (including those with challenges such as Sanfilippo to attend – we needed to be away for 5 days at least from the UK to be able to see the core conference material). It wasn’t something I would choose to do ideally…We felt it was an essential thing to do as a newly diagnosed family considering trial options to get access to the latest information and ask questions directly to experts in poster sessions. It was invaluable for us.
- We are in Australia. I have 2 children with attenuated Sanfilippo type A (as well as a newborn). Attending would be a near impossibility for our family. Both the cost and difficulty travelling that far (13 hours just to get to LAX).
- I am very grateful indeed for the free registration for family members! We live in Spain and it would have been almost impossible for us to attend the congress otherwise.”
Cure Sanfilippo Foundation seeks to support research that fills critical gaps in current knowledge across basic science, clinical care, and translational therapeutics which will ultimately improve the lives of children with Sanfilippo syndrome. Scientist-clinician-patient collaborations are highly encouraged. The Foundation is available to assist in facilitating connections with patients/caregivers to inform projects as needed.
Sanfilippo syndrome (MPS III) is a multisystem metabolic disease with prominent neurodevelopmental and neurodegenerative consequences for affected children. There are currently no treatment options available for this terminal condition but we are working hard with the scientific community to change that!
Deadline to Apply: Submit your Letter of Intent (LOI) using the downloadable form to: Research@nullCureSanfilippoFoundation.org,
For more information on this current funding opportunity and LOI form, we invite you to visit us at:
For additional questions, contact Cara@nullCureSanfilippoFoundation.org.
Recent research in Spain suggests that home-based infusion may be necessary to minimize patient exposure to COVID-19 in the clinic. The full text of the article, as published in the journal Blood Cells, Molecules & Diseases , is available here.
The National Gaucher foundation has provided COVID-19 tips, precautions, and a COVID-19 glossary as well as other resources, on their website for Gaucher patients and families.
The National Tay-Sachs and Allied Diseases (NTSAD) Association has released a statement on their website discussing their ongoing operations and available patient resources during the COVID-19 crisis.
The Fabry Support & Information Group (FSIG) features an Appointment Companion portal on its website – to help patients remotely inform Fabry care teams during the COVID-19 crisis about their treatment goals and challenges, browse clinical trials, and compare treatment centers.
The National MPS Society USA has posted recommendations for patients and families concerned about COVID-19.
On Oct. 3, 2019, NIH announced awards to expand the RDCRN.
The RDCRN is designed to advance medical research on
rare diseases by providing support for clinical studies and facilitating
collaboration, study enrollment and data sharing.
Currently, the RDCRN consists of 20 distinct clinical research consortia and a Data Management and Coordinating Center (DMCC). Each consortium focuses on at least three related rare diseases, participates in multisite studies and actively involves patient advocacy groups as research partners. The DMCC enables uniform high-quality data collection and analysis and facilitates information sharing across the network. This robust data source helps scientists better understand the common elements of rare diseases so they may apply that knowledge to improving diagnosis and treatment for these conditions.
Lysosomal Disorders Network headed by Chester B. Whitley, M.D., Ph.D., University of Minnesota, Minneapolis was the recipient of one of the awards.
Learn more about this project in NIH RePORTER.