New funding paves the way for novel LAMA2-CMD therapeutic research
Stichting voor Sara and Prinses Beatrix Spierfonds Join Forces for Promising Muscular Disease Research
Stichting voor Sara and the Prinses Beatrix Spierfonds are collaborating to fund an innovative study into a new therapetuic approach for LAMA2-CMD. The goal of this research is to halt the progression of the disease in affected individuals. Within two years, the researchers aim to map out a path towards this potential therapy.
Research Led by Dr. Boris Shneyer and Prof. Bert Smeets
The study, being conducted at Maastricht UMC+, is led by Dr. Boris Shneyer under the direction of Prof. Dr. Bert Smeets. The approach is similar to the existing exon-skipping therapy for Duchenne muscular dystrophy, which means the development of the therapy could be accelerated once successful results are achieved in the laboratory.
LAMA2-CMD: A Rare and Debilitating Disease
People with LAMA2-CMD experience reduced muscle tone and weakness. Most patients are unable to walk independently and face difficulties with both gross and fine motor skills. They may also encounter problems with their respiratory and swallowing muscles, often requiring breathing assistance, particularly at night. Children with this disease typically do not live to adulthood, and the muscle weakness severely limits their ability to function in daily life.
“This research brings hope to patients and their families,” says Bram Verbrugge from Stichting Voor Sara. “It is incredibly valuable that we can join forces with the Spierfonds. We are extremely grateful for the contributions from both organizations to this study. Through this collaboration, we can take faster steps towards an effective treatment.”
Financial Support for the Research
This groundbreaking research is made possible by the funding provided by Stichting Voor Sara and the Spierfonds, with additional contributions from the Pieter Bastiaan Fund and Stichting Zabawas.
In addition to this collaboration, the Spierfonds and Stichting Voor Sara are also working together on natural history research into this rare disease. Conducted at RadboudUMC, this study tracks the progression of LAMA2-CMD over time in Dutch patients. This will improve patient care and pave the way for clinical trials. The data gathered from this research can also be used to prove the effectiveness of future treatments.
The Prinses Beatrix Spierfonds is a Dutch charity dedicated to supporting research into muscle diseases, providing funding for innovative studies aimed at improving the lives of people affected by these conditions. Through this new collaboration with Stichting Voor Sara, the Spierfonds is helping to fund groundbreaking research into new therapies for Lama2-CMD.
