In the ever-evolving field of rare disease research, it's crucial to remain informed about the latest progress. At Sano, our commitment is to stay ahead of these developments. We are excited to offer a curated overview of some of the most significant advancements in rare disease research from the past few months.
US FDA approves two gene therapies for sickle cell disease: Groundbreaking therapies for sickle cell disease, including a CRISPR-based gene editing treatment, have been approved, offering hope to over 100,000 Americans.
FDA issues final guidance on rare disease drug development: The FDA has issued final guidance emphasising flexibility and patient focus in rare disease drug development, aiming to enhance the efficiency and efficacy of treatments for severe conditions.
mRNA therapy shows promise for curing children's rare liver disease: An mRNA therapy, effective in treating argininosuccinic aciduria in mice, has shown promise for human treatment, potentially curing this and other genetic diseases.
Groundbreaking online community connects Progeria patients worldwide: A unique online platform, Progeria Connect, launched to enable global connection and support for individuals with Progeria, enhancing access to medical information and emotional support.
UB research identifies key mechanism in infantile cystinosis: University at Buffalo research identified a key mechanism in infantile cystinosis, suggesting CRISPR genome-editing as a potential cure.
Pilot launched to support children with rare conditions to access personalised therapies: The UK government's Rare Therapies Launch Pad program launched to focus on developing access to personalised therapies for children with rare diseases.
Arrowhead Pharmaceuticals presents plozasiran and zodasiran data at AHA 2023: Arrowhead Pharmaceuticals presented Phase II clinical data for RNA-based therapies plozasiran and zodasiran, targeting lipid metabolism disorders, highlighting significant potential in treating severe hypertriglyceridemia and mixed dyslipidemia.
These achievements highlight the commitment of the rare disease community to expanding knowledge and finding solutions. At Sano, we are devoted to driving advances in rare genetic disease research. Our expertise includes participant recruitment, genetic and biomarker testing, and fostering long-term participant engagement. Contact us below to learn more.