Rare cancers account for almost a quarter of all new cancers worldwide though there is no universally adopted definition for rare cancers. In the U.S., rare cancers are defined as those with fewer than 15 cases per 100,000 per year,...
Expertise: Rare Disease 87 results
Rare Disease
Premier Presentations from WODC
Watch Premier Research’s presentations from World Orphan Drug Congress 2020 below. Note: You must be logged into your WODC Swapcard account to view presentations Rare Disease Recruitment: Addressing Enrollment Hurdles and Differentiating Your Clinical Trial in an Increasingly Competitive Research...
Between 2010 and 2018, 33.8 percent of the 402 orphan indications approved by the FDA were approved for children only or targeted pediatric diseases.[1] The rare pediatric disease priority review voucher program is one incentive that’s helped to spur research...
Gene therapy products are typically designed to achieve therapeutic effect through long-acting or permanent changes in the human body. Due to this extended period of exposure, patients who receive gene therapies may be at increased risk of delayed adverse events....
Despite the passage of various legislative initiatives aimed at increasing the frequency and quality of pediatric clinical trials, neonates continue to be treated with medicines that have not been studied or approved in this population.1 Historically, sponsors may have felt...
Rare Disease
The State of Cell Therapies in Dermatology
Skin contains essential stem cell populations and other cell types that are critical for renewing and maintaining its structural integrity and function.1 Of the many types of skin stem cells that have been identified, epidermal stem cells — primarily keratinocyte...
Rare Disease
The State of Gene Therapy in Dermatology
When it comes to dermatologic conditions, gene therapy is still in its very early stages. However, we are seeing promising potential solutions for some rare genetic dermatology diseases, as well as ongoing research in more common skin conditions. The success...
Background With their potential for long-term or even curative efficacy, gene therapies are of great interest to researchers, clinicians, patients and caregivers alike. But coordinating and conducting a global, multi-center gene therapy trial is a complex, high-risk undertaking. Beyond the...
Gene therapy holds the potential of long-term efficacy or even a cure for diseases with an underlying genetic basis, offering hope to patients with currently incurable diseases. The use of gene therapy for dermatologic conditions is attractive for many reasons:1...
At Premier Research, we attend a lot of events throughout the year – and through these events, we have the opportunity to meet individuals from all sides of the clinical development process. At the recent Global Genes RARE Patient Advocacy...