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The current drug development framework was not designed for #ultrarare diseases. We cannot afford to let diseases go untreated – and to let children continue to die – when the science to provide a treatment exists. Join us at?www.ActforUltrarare.com?to help create an ecosystem that will stimulate development of treatments for ultrarare diseases. #ActforUltrarare
Scott Schliebner
2 周
I registered and look forward to learning more.
Kaye Spratt
2 周
This is great! I registered.
Biotech & Healthcare Executive | Medical Strategy Expert | Med Affairs Innovation | Clin Dev & Market Access | RWE & HEOR | | Cell & Gene Therapy | Transforming Complex Challenges into Scalable Success
2 周Registered, looking forward to it! You are right: the current system was not designed for gene therapies nor for extremely small patient populations. We need innovative approaches to clinical trial design, regulatory pathways, and reimbursement models tailored to gene therapies for ultrarare diseases. Ultragenyx's recent progress with UX111 for Sanfilippo shows the great potential of gene therapies in rare disease.?To accelerate development, we should consider accepting alternative trial designs and biomarkers as primary endpoints. We also need newborn screening for these conditions. As a med affairs professional, I believe these changes are essential to making gene therapies a reality for patients and families.