Publication highlights the unique challenges of treating an extremely rare patient population and the solution available today to provide hope to these patients
SAN DIEGO–(BUSINESS WIRE)–#ASO—n-Lorem, a nonprofit foundation, today announced the publication of a review article titled “Meeting the Needs of Patients with Ultra-rare Diseases” in Trends in Molecular Medicine (Crooke, S.T., Trends in Molecular Medicine, Advanced Online Publication, January 2022). In this publication, Dr. Crooke outlines the unique challenges of developing potentially life-saving treatments for a small number of ultra-rare patients, who because of their unique genetic mutation, are only one of a few patients worldwide. To distinguish this extremely small patient population, n-Lorem refers to these patients as nano-rare patients (1 to 30 patients worldwide). n-Lorem is the only non-profit foundation employing a charitable model that can meet the challenges of some of these nano-rare patients. n-Lorem uses a robust and proven ASO technology to discover and provide experimental antisense oligonucleotide (ASO) medicines for nano-rare patients for free, for life.
“Today, we have all the necessary components to tackle the previously insurmountable challenge of developing personalized treatment options for nano-rare patients,” said Stanley T. Crooke, M.D., Ph.D., Founder, CEO and Chairman of n-Lorem Foundation. “Genetic characterization is feasible and becoming more broadly accessible, which enables more patients to seek out specialty physicians at institutions who are best equipped to support them. More than thirty years of investment in ASO technology provides the opportunity to discover and develop an optimized experimental medicine targeted to a unique, single genetic mutation, and regulatory guidance provides a supportive environment to develop ASO therapeutics for these patients.”
“Today, we are making a difference, but we are not doing it alone. We are fortunate to have the trust of the patients and their families, the dedication of an esteemed group of physicians, the support and resources of the ultra-rare disease communities and patient advocacy groups, and the commitment and financial support of organizations across all areas of drug discovery, development and manufacturing,” continued Dr. Crooke.
“We have assisted in the development and treatment of 14 nano-rare patients and received over 100 applications for treatment with more than 40 nano-rare patients approved. We can do more, and we are creating the broadest possible network of donors and collaborators to support that. We hope that this publication highlights the collaborative approach necessary to address the desperate need of these patients,” concluded Dr. Crooke.
- Watch: n-Lorem Foundation Delivers Hope and Treatment for Patient with Nano-Rare Disease
- Watch: n-Lorem Foundation: Creating a Better Future for Nano-Rare, One Patient at a Time
n-Lorem Foundation is a non-profit organization established to apply the efficiency, versatility and specificity of antisense technology to charitably provide experimental antisense oligonucleotide (ASO) medicines to treat nano-rare patients diagnosed with diseases that are the result of a single genetic defect unique to only one or very few individuals. Nano-rare patients describe a very small group of patients (1-30 worldwide) who, because of their small numbers, have few if any treatment options. n-Lorem Foundation was created to provide hope to these nano-rare patients by developing individualized ASO medicines, which are short strands of modified DNA that can specifically target the transcripts of a defective gene to correct the abnormality. The advantage of experimental ASO medicines is that they can be developed rapidly, inexpensively and are highly specific. To date, n-Lorem has assisted in the development and treatment of 14 nano-rare patients and received over 100 applications for treatment with more than 40 nano-rare patients approved. n-Lorem was founded by Stanley T. Crooke, M.D., Ph.D., former chairman and CEO of Ionis Pharmaceuticals, who founded Ionis Pharmaceuticals in 1989 and, through his vision and leadership, established the company as the leader in RNA-targeted therapeutics. Follow us on Twitter, Facebook, LinkedIn and YouTube.
Amy Williford, Ph.D.
Director of Communications