DEPARTMENT OF HEALTH & HUMAN SERVICES
National Institutes of Health
Bethesda, Maryland 20892
Dear Workshop Participants,
The National Institute of Neurological Disorders and Stroke (NINDS) and the Office of Rare Diseases (ORD) at NIH along with the International Society for Mannosidosis & Related Diseases, Inc. (ISMRD) are sponsoring a workshop, “The Glycoproteinoses: An International Workshop on Advances in Pathogenesis and Therapy.” On behalf of the co-organizers (Drs. Alessandra d’Azzo, Leena Peltonen and Steven Walkley, I would like to invite you to participate in this important workshop which is the first to be held for this group of disorders. The workshop will take place on April 1-2, 2004 at the Double Tree Hotel and Executive Meeting Center in Rockville, Maryland.
The workshop is intended to address the following key questions and issues:
· What is the pathomechanism on how defects in processing glycoproteins (as opposed to glycoaminoglycans) in the endosomal-lysosomal system lead to disease?
· What are the commonalities and differences for each disease type?
· Can the success in ERT and CMT treatment, particularly for a-mannosidosis, be translated to other related disorders?
· How should assessments be conducted for any proposed treatment(s)?
The goals of this workshop include:
· To provide scientists and clinicians working or interested in these group of disorders an opportunity to gather and to discuss avenues for medical and research breakthroughs
· To facilitate data-and resource-sharing amongst investigators, including newly characterized animal models
· To foster collaborations amongst scientists and interactions with patients and families
The workshop is organized into 6 sessions, each consisting of short scientific presentations by invited speakers, with each session followed by a panel discussion (please refer to the attached Meeting agenda for session topics and presenters). A summary of the panel discussion from each session will be presented during the final session of the workshop along with recommendations of priorities for research and resources.
Registration and logistical information regarding travel and lodging for the workshop are enclosed with this letter. MasiMax Corporation is providing the logistical support for this meeting for NINDS. Please respond by February 17, 2004 to Ms. Rechá Bullock-Jones at (240) 683-1743 or by e-mail at regarding your participation in this workshop.
A limited amount of support is available for junior investigators to participate and to present a poster during the workshop. We are asking that you nominate a junior investigator from your laboratory and submit an abstract (450 words or less) of the work to be presented. These abstracts should be submitted directly by e-mail to Dr. Steven Walkley () no later than February 20th. The organizers of the workshop will make the selection based on the information provided, and award travel and lodging support to the successful nominees. We will also want invited speakers to submit abstracts for their own presentations as well as for poster presentations from their lab. We request that these abstracts be submitted directly by e-mail to Dr. Alessandra d'Azzo () no later than March 1st.
Please note that a Family Conference organized by ISMRD will commence at the conclusion of the scientific workshop. Information regarding participation on the Family Conference can be obtained at http://www.mannosidosis.org.
On behalf of the organizers, I look forward to your participation.
Sincerely yours,
Danilo A. Tagle, Ph.D.
Program Director in Neurogenetics
National Institute of Neurological Disorders and Stroke, NIH
6001 Executive Blvd., Neuroscience Center, Rm 2133
Bethesda, MD 20892
301-496-5745