U.S FDA designated Aytu BioPharma’s Vascular Ehlers Danlos Syndrome treatment ‘AR101’ as a fast track. The treatment had already been designated as an orphan drug in the United States and Europe. The company will conduct a clinical trial in 260 COL3A1-positive VEDS patients.
A research paper has been published that analyzes the reasons for a delay in the diagnosis of rare diseases and suggests methods for improvement. Researchers at Osaka University took a qualitative approach by interviewing patients with hereditary angioedema (HAE). Researchers have concluded that it is vital to raise awareness of rare diseases for both patients and clinicians to prevent delays in diagnosing rare diseases.
Sharing health data is critical to advancing rare disease research and improving clinical care. The European Commission will propose a governance framework that will enable safe and efficient sharing of health data between health systems within Europe through the European Health Data Space (EHDS).
At the 2022 annual meeting of the American Academy of Neurology, it was announced that clinicians should consider disease-modifying strategies when treating patients with rare genetic disorders. Professor Lisa Emrick says that clinicians need to think about gene-based therapy and treatment based on the genetic cause of the disease.
Rare disease pharmaceutical industry trendsIonis Pharmaceutical and AstraZeneca announced that they had succeeded in Phase 3 clinical trial of co-developing transthyretin amyloidosis treatment(eplontersen). However, the…