n-Lorem Foundation’s Nano-rare Patient Colloquium Draws More Than 750 attendees, Reinforcing Need for Treatment Options for People with Nano-rare Illnesses

n-Lorem has made tremendous progress over the last five years to discover, develop and provide optimized, personalized ASO medicines to nano-rare patients and provide them for free, for life.

The colloquium united and informed members of the nano-rare community, with attendees representing more than 25 U.S. states and territories and over 20 countries worldwide.

n-Lorem, a nonprofit foundation, drew more than 750 researchers, scientists, healthcare providers, patients and their families to its second annual Nano-rare Patient Colloquium hosted by Biogen. The two-day event in Cambridge, MA, united supporters of n-Lorem's mission, amplified the voices of nano-rare patients, and informed the community on the remarkable observations in n-Lorem treated nano-rare patients. To date, treatments have been initiated for more than 20 patients, with an additional patients expected to begin treatment by year’s end.

“This is the second year of our colloquium and, while the first year was incredibly successful, the year was even better than we could have predicted with overwhelming positive responses from participants and attendees," said Stanley T. Crooke, M.D., Ph.D., Founder, Chairman and CEO, n-Lorem Foundation. “This year, we shared encouraging positive data on a number of patients who have been on treatment for a year or more, and early clinical data on patients who began treatment this year. These patients are experiencing remarkable positive changes in multiple domains, such as walking, putting on their shoes and recovery of emotional and behavioral attributes. We believe that this is just the beginning for what we can do for nano-rare patients.”

In addition to 40 panelists and presenters, more than 175 nano-rare patients and family members joined the colloquium. Registration grew by nearly 50 percent compared to 2023, including more than 150 scientists, researchers and healthcare providers, and a 100 industry affiliates and partners in attendance. All presentations and panel discussions are available for viewing on the n-Lorem website.

“Today, our only barrier to providing treatments to more nano-rare patients is funding,” continued Dr. Crooke. “I urge organizations and individuals to support our mission and help us discover, develop and provide the most optimal ASO medicines we can for even more nano-rare patients.”

To date, n-Lorem has evaluated more than 300 applications for treatment and accepted more than 140 nano-rare patients into its program. n-Lorem has filed more than 20 investigational new drug (IND) applications and has initiated treatment in more than 20 patients.

Donations to help support n-Lorem’s ongoing work can be made through the foundation’s website: https://www.nlorem.org/donate/.

About n-Lorem

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 received over 300 applications for treatment with more than 140 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.

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

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