n-Lorem, a nonprofit founded by Dr. Stanley Crooke, develops antisense oligonucleotide therapies for patients with nano-rare diseases, including KIF1A-associated neurological disorder (KAND). The organization provides these treatments free for life, addressing a critical gap in care for patients with rare genetic mutations.
Actor turned rare disease advocate Luke Rosen tells how his daughter, Susannah, lives with a KIF1A-associated neurological disorder, or KAND. Launched in 2020 by Ionis Pharmaceuticals founder and CNBC Advisory Board member Dr. Stanley Crooke, n-Lorem is a nonprofit organization that develops antisense oligonucleotide, or ASO, therapies for patients with nano-rare diseases and provides the treatments to the patients for free for life. At the time of Susannah's diagnosis, in 2016, there were no treatments for KIF1A, and no clinical trials underway or literature to lean on for answers. Susannah Rosen at the hospital for her ASO treatment. ... "The FDA defines rare disease as a patient population of 200,000," Crooke said in an interview with CNBC. "But we now know that there are many, many pathogenic mutations that produce disease in far fewer patients ... Patients with the same mutation can be treated with the same medicine, but if n-Lorem needs to develop a new treatment, the average cost is $1.2 million, he said. Since the foundation's launch, it's had more than 400 rare disease patient applicants, of which it has been able to accept about 200, Crooke said.
Original title: “Rare disease treatment: New drug fights neurological gene mutation”