Solve FSHD has provided one million dollars in support of n-Lorem Foundation’s efforts to fully understand the complex molecular cause of facioscapulohumeral muscular dystrophy-2 (FSHD2). This research will be critical to identifying a strategy for treatment for FSHD2 patients. In addition, this funding supports the expansion of n-Lorem’s research and discovery efforts, significantly enhancing the capabilities of the foundation.
“We are grateful to Solve FSHD for this generous support. Not only do we hope to better understand FSHD2, but we hope to identify a path forward for a therapeutic strategy for these patients,” said Stanley T. Crooke, M.D., Ph.D., Founder, CEO and Chairman of n-Lorem Foundation. “Additionally, as we invest in expanding our research capabilities, we enhance the efficiency and capacity of our drug discovery and development programs, which will benefit all of our patients today and in the future.”
FSHD2 is a form of muscular dystrophy that affects approximately five percent of FSHD patients. FSHD2 has been linked to mutations on a gene called SMCHD1, but other FSHD2 genes have been identified and approximately two percent of FSHD cases have an unknown genetic cause. The molecular complexity of FSHD2 makes the development of an effective treatment extremely challenging.
“We are pleased to be partnering with n-Lorem Foundation to gain further insight into the molecular mechanisms that lead to FSHD2. Until the molecular cause of FSHD2 is better understood, progress on a potential therapeutic is greatly disadvantaged. n-Lorem is the only non-profit with access to three decades of learning in the RNA-targeted therapeutic space and the scientific team to aggressively pursue this complex disease,” said Dr. Eva Chin, Executive Director of Solve FSHD.
“Our hope is that this funding will further our understanding of FSHD2, but also other programs, patients and their families,” added Chip Wilson, Founder and Chairman of the Board for Solve FSHD. “We are extremely grateful for the pioneering work of Dr. Crooke and his talented team of scientists at the Foundation.”
[Source(s): n-Lorem Foundation, Business Wire]