Advancing Breakthroughs for Rare Disease Therapies

With over 7,000 rare diseases impacting more than 350 million people worldwide, there is a growing need to develop safe and affordable therapies for those affected. In honor of Rare Disease Month (February) and Rare Disease Day (February 29), our commitment to advancing therapies for rare diseases and advocating for a brighter future remains at the forefront of our mission. Learn more > 

Case study: Pursuing Breakthrough Therapies for Rare Metabolic Diseases

A biotech company pioneering gene therapies for metabolic diseases partnered with Veristat to support their Phase I/II clinical study for the treatment of a rare human genetic lysosomal storage disorder. Veristat’s Full-Service clinical trial team helped tightly coordinate the gene therapy’s dosing administration and data review while overcoming critical patient, lab, and manufacturing logistics challenges. Find out more >

Podcast: Clinical & Regulatory Considerations for Developing Rare Disease Treatments

When developing a novel rare disease therapy, adopting a strategic approach early in the development process supports a well-designed study and reduces regulatory risk along the way.  Listen to our podcast as Veristat experts share the clinical and regulatory challenges of developing rare disease products and key considerations for planning studies in the US and globally. Learn more >

Blog: FDA CDER’s Accelerating Rare Disease Cures (ARC) Program

In 2022, CDER launched the ARC Program to expedite the development of therapies for rare diseases by promoting scientific innovation, regulatory understanding, and collaboration with stakeholders to address the complexities of rare disease drug development. Read our blog to learn more about the program’s annual initiatives and goals. Read now >

Infographic: FDA Expedited Pathways

Veristat Regulatory experts break down the benefits of, qualifying criteria for, and timing and procedures for six different expedited programs and designations. Do you want to save time and reduce costs? See which of the FDA’s different programs you can qualify for. Learn more >

Blog: Cycle for Survival 2024

For the tenth consecutive year, Veristat participated in Memorial Sloan Kettering's Cycle for Survival, successfully raising over $90K to support rare cancer research. At Veristat, we stand united in championing a better tomorrow. Read the Blog >