Dr. Florian Eichler, Massachusetts General Hospital Center for Rare Neurological Diseases, Boston, MA
Dear CRF Families and Adults with Cystinosis,
We are excited to let you know that our CRF funded study “Clinical Trial Readiness for Distal Myopathy in Nephropathic Cystinosis” has resulted in a clinical trial! The title of the clinical trial is “Distal Myopathy and Muscle Wasting in Nephropathic Cystinosis”. We have good news to share – the study has been extended. We are close to the full enrollment of 20 patients but need a few more patients for the study.
If you’re interested in participating, please contact research coordinator Rachel Duong at 617-724-1330 or email@example.com. The clinical trial is open to all adults with cystinosis 18 years or older. This study will help us learn more about how cystinosis progresses and how it varies within the cystinosis population. It is an extremely important study and will help us develop a plan to treat myopathy.
We hope that you will want to volunteer and be part of the study. We have attached the recruitment letter for the study. Clink here for the PDF with more information. Please contact Rachel Duong, to participate via phone at (617) 724-1330 or by email at firstname.lastname@example.org. We need your help so please spread the word to other cystinosis families and patients!
Dr. Paul Goodyer, McGill University Children’s Hospital
Dear Cystinosis Patients and Families:
We would like to inform you about a new study conducted by Dr. Paul Goodyer at McGill University Children’s hospital in Montreal. Dr. Goodyer is conduction a research study to better understand the spectrum of genetic mutations that lead to cystinosis (Cystinosis Nonsense Mutation Screen-CyNoMuS). He is seeking volunteers who would like to know whether or not they carry a CTNS nonsense mutation.
If you are interested and want to learn more about the study, please read the PDF flyer and contact them by phone at (514) 412-4400 ext. 22953 or by email to Murielle M. Akpa, PhD, email@example.com
The following studies are being funded by the CRF to advance knowledge in key areas of cystinosis treatment. Click the following links to see the full information for each study.
Volunteers Needed for a Research Study of Bone Health and Muscle Strength – Children and adults with cystinosis are at risk for musculoskeletal disease. This research study, performed by Dr. Mary Leonard and Dr. Paul Grimm, will be the first comprehensive evaluation of bone structure and muscle strength in children and adults with cystinosis.
Research Study of Bone Marrow Transplant for Cystinosis Patients from Matched Related Donor – The clinical research study is being conducted at the Ronald Reagan UCLA Medical Center.