09 May 2020
CDLS - Hope for Hasti partners with the jackson laboratory
On the scientific front, there has been some great news!
CdLS Hope for Hasti is now working in partnership with the amazing Jackson Laboratory. JAX have been conducting cutting-edge research for 90 years. They have more than 70 research teams and an astonishing 26 Nobel Prizes are associated with their research. We at CdLS Hope for Hasti are absolutely delighted to be working in partnership with the very best in this field.
Research by Lutz and her collaborators at JAX led to the development of Spinraza®, the first FDA-approved drug to treat spinal muscular atrophy (SMA), a rare neurodegenerative disease that affects children.
See here more about the work JAX does on rare disease. Jackson Laboratory Rare and Orphan Disease Centre.
Having JAX on board working on this project gives our donors and grant providers reassurance that creating a treatment for CdLS is a viable project and gives us huge confidence that we are on the right track. All our consultations with JAX have been positive and they have high hopes for our project.