Funded Rare Disease Clinical Research Training Program

“Rare Opportunity for Young Researchers”

The Rare Diseases Clinical Research Network (RDCRN) annually offers early-career rare disease researchers this excellent training and networking opportunity. This is available to researchers associated with one of the RDCRN’s research consortia. (The Lysosomal Disease Network is one such RDCRN consortium.) The Rare Diseases Training Program is recruiting for the 2019 – 2020 academic year. More information about the Rare Diseases Training Program, as well as the form to complete and submit as part of your application, are available by clicking on the download link on this web page (scroll down). The Rare Disease Clinical Research Training Program accepts applications and sends-out acceptances on a rolling basis.

This academic-year-long course consists of both in-person and remote sessions providing the tools and mentoring needed for a successful career in rare diseases clinical research. Topics include: statistics in small populations, interactions with industry and pharmaceutical companies, successful grant writing, working with patient and family advocates and groups, research career design and planning, and successful publishing in the field of rare diseases.

Participation requires a 10% time-commitment from the applicant’s program chair or department chair for the applicant’s dedicated, focused research time. In addition, the program requires: two 2-day trips to the Washington, D.C. area; two 1-hour webinars per month; completion of a mentored research project with culmination in a poster presentation at the Fall 2020 RDCRN meeting in the Washington, D.C. area; and about 4 hours of projects/reading per month for preparation. Certificates will be awarded to participants completing at least 75% of the course content at or above acceptable levels. The first 2-day trip to the Washington, D.C. area will occur October 21-22, 2019.

Twenty competitive participants will be awarded a “funded” position. This funding will provide $1,000/year reimbursement towards attending the two 2-day trips to the Washington, D.C. area. Applicants can apply for the funded program (the “travel group”), while also indicating that they wish to be considered for the unfunded program if not chosen for funding. Up to twelve additional unfunded positions are available to excellent applicants this year.

Applicants are requested to submit the following: the completed downloadable application form (scroll down); their CV; and a letter of commitment from their supervisor supporting their participation in this Training Program, as well as offering to support any costs that exceed the reimbursed costs (if any) of participation in this Training Program.

Join the Contact Registry

You (or your child) are invited to participate in the nationwide Contact Registry for lysosomal disease patients. The Rare Diseases Clinical Research Network (RDCRN) offers this patient Contact Registry. This is a method by which patients with lysosomal diseases can register themselves online with the RDCRN in order to be contacted in the future about clinical research opportunities and updates on the progress of the research projects. The contact registry is anonymous and free of charge.

The Lysosomal Disease Network encourages lysosomal disease patients or their families to register. Because of the rarity of lysosomal diseases, the Contact Registry plays an important role in estimating the incidence and prevalence of these conditions, and in keeping interested persons informed about available clinical research for treatment and cure of lysosomal

Conference on Clinical Research in Rare Diseases

Conference on Clinical Research in Rare Diseases Presented by the Rare Diseases Clinical Research Network


Research is the Only Way Forward

The Rare Diseases Clinical Research Network is hosting the 4th Conference on Clinical Research in Rare Diseases at the Washington Marriott Wardman Park Hotel in Washington, D.C. on Thursday, November 3, 2016. This unique conference will focus on research methodology for rare diseases, and is of particular interest to trainees, fellows, and junior faculty engaged in research in rare diseases. For more information about the Conference, including details about the program, how to submit your abstract, how poster-presenters can apply for travel awards, and Conference logistics and registration, visit the Conference website: 4th Conference on Clinical Research in Rare Diseases. Attendees must register for the meeting. There is a meeting registration fee of $100, which includes meeting materials, breakfast, lunch, snacks and refreshments. The registration deadline is October 19, 2016. Abstracts will be accepted through October 3, 2016. Much more information is available via the registration page, so please become informed now!

The Lysosomal Disease Network, which is part of the Rare Diseases Clinical Research Network (RDCRN), will be well represented at the Conference, along with all the other RDCRN rare diseases research consortia. As a rare disease researcher, it is not required that you be a member of one of the RDCRN rare diseases consortia in order to attend this Conference — all interested rare disease researchers are welcome, and encouraged, to register and attend. Please join us!