Working Towards an ASO Treatment for FSHD2 Patients

Budget

US$700,000 

Lay Abstract

Investigator: Dr. Stanley Crooke

FSHD is a rare disease. FSHD2 is even more rare. Only 5% of all FSHD patients have FSHD2. We don’t want them to get left behind.

The n-Lorem Foundation is a non-profit organization established to charitably provide experimental antisense oligonucleotide (ASO) medicines to treat patients with extremely rare diseases like FSHD2. The advantage of ASO medicines is that they can be developed rapidly, inexpensively and are highly specific.

This is the first step of a research project aimed at understanding the molecular cause of FSHD2, which once complete will inform therapeutic strategies for patients with FSHD2. Several leading FSHD researchers are providing the overall guidance on this project.

The cost of this first step is US$700,000. It covers the purchase and installation of specialized laboratory equipment. After the equipment and training are in place — the work to better understand FSHD2 can begin.

The FSHD Canada Foundation acknowledges the generous support of the Wilson 5 Foundation towards the funding of this grant.