SOLVE FSHD Provides Funding to n-Lorem for Collaboration to Expand Understanding of FSHD2, a Rare Form of Muscular Dystrophy

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SAN DIEGO–(BUSINESS WIRE)–n-Lorem, a nonprofit foundation, today announced that Solve FSHD has provided $1 million to support n-Lorem’s efforts to fully understand the complex molecular cause of Facioscapulohumeral Muscular Dystrophy-2 (FSHD2). This research will be essential to identify a treatment strategy for FSHD2 patients. Additionally, this funding supports the expansion of n-Lorem’s research and discovery efforts, greatly enhancing the foundation’s capabilities.

“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 way forward for a therapeutic strategy for these patients,” said Stanley T. Crooke, MD, Ph.D., Founder, CEO and Chairman of the n- Lorem. . “Additionally, as we invest in expanding our research capabilities, we are improving the efficiency and capacity of our drug discovery and development programs, which will benefit all of our patients today and in the future. ‘coming.

FSHD2 is a form of muscular dystrophy that affects approximately 5% of patients with FSHD. FSHD2 has been linked to mutations in a gene called SMCHD1, but other FSHD2 genes have been identified and about two percent of FSHD cases have an unknown genetic cause. The molecular complexity of FSHD2 makes developing an effective treatment extremely difficult.

“We are delighted to partner with the n-Lorem Foundation to better understand 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 nonprofit 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.

“We hope this funding will deepen our understanding of FSHD2, but also other programs, patients and their families,” added Chip Wilson, Founder and Chairman of the Board of Solve FSHD. “We are extremely grateful for the pioneering work of Dr. Crooke and his talented team of scientists at the Foundation.

Learn more about n-Lorem’s mission at www.nlorem.org and consider donating to n-Lorem to bring hope, possibility and treatment options to these patients and families in need.

  • Subscribe: n-Lorem Patient Empowerment Program Podcast Series
  • Watch: The n-Lorem Foundation offers hope and treatment to patient with nano-rare disease
  • Watch: n-Lorem Foundation: Creating a brighter future for nano-rare, one patient at a time

SOLVE FSHD

Solve FSHD is a Vancouver-based philanthropic venture capital organization created to catalyze innovation and accelerate key research in the search for a cure for Facioscapulohumeral Muscular Dystrophy (FSHD). FSHD is one of the most prevalent forms of muscular dystrophy, and the organization will work as a catalyst to accelerate drug development and treatments for FSHD2 while further benefiting FSHD1. Founded by renowned Canadian entrepreneur and philanthropist Chip Wilson, founder of technical apparel company lululemon athletica inc. Chip has committed $100 million to kickstart funding for projects that support the organization’s mission to find a cure for FSHD by 2027.

Solve FSHD’s goal is to find a solution that can stop muscle degeneration, increase muscle regeneration and strength, and improve the quality of life for people with FSHD. The organization will fund innovative biotechnology and biopharmaceutical research and development activities that accelerate new treatments and therapies for FSHD.

About n-Lorem

The n-Lorem Foundation is a nonprofit organization created to apply the efficacy, versatility, and specificity of antisense technology to provide charitable investigational antisense oligonucleotide (ASO) drugs to treat diagnosed nano-rare patients. with diseases resulting from a single genetic defect unique to one or very few individuals. Nano-rare patients describe a very small group of patients (1 in 30 worldwide) who, due to their small numbers, have few or no treatment options. The n-Lorem Foundation was created to bring hope to these nano-rare patients by developing individualized ASO drugs, which are short strands of modified DNA that can specifically target the transcripts of a faulty gene to correct the ‘anomaly. The advantage of ASO investigational drugs is that they can be developed quickly, inexpensively and are highly specific. To date, n-Lorem has assisted in the development and treatment of 14 nano-rare patients and has received over 150 treatment requests with over 70 nano-rare patients approved. n-Lorem was founded by Stanley T. Crooke, MD, Ph.D., former President and CEO of Ionis Pharmaceuticals, who founded Ionis Pharmaceuticals in 1989 and, through his vision and leadership, has makes the company the leader in therapeutics. follow us on TwitterFacebook, LinkedIn, YouTube and Instagram.

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