Certain studies conducted by Engage Health are closed, and recruitment is targeted to certain individuals. Other studies are open, and allow recruitment from a number of sources.
The following are current market research projects that are open to participation.
Cystinosis Patients—Free Genetic Testing for Nonsense Mutation
McGill University invites cystinosis patients to join their study concerning a specific type of genetic alteration called a “Nonsense Mutation”. Although cystinosis is caused by many different disruptions of the CTNS gene, they are particularly interested in “nonsense mutations” which trick the cell into stopping production of Cystinosin protein. Several pharmaceutical companies are working hard to develop medications related to a well-known antibiotic (gentamicin) that permits the cell to disregard nonsense mutations. To participate in the free genetic testing study and learn if you have this nonsense mutation, contact the study coordinator, Murielle Akpa, PhD either by phone or email; phone: (514) 412-4400 ext. 22953 email: email@example.com.
Our client is interested in learning more from physicians about their cystinosis patients and their interest in clinical trials. To denote your interest and share your experiences, please click this link.
US EB Patients- Health Research Telephone Interview- $100 Honorarium
Engage Health has been retained by a client who is working on an investigational agent in Epidermolysis Bullosa (EB). We are conducting a health research study with adult patients or parents of minor patients in the US who have EB. The objective of this research is to better understand the issues that are faced by patients with EB, including the issues around current therapies. The research consists of an approximate one hour telephone interview that will be conducted by a trained interviewer. Individuals who complete the interview will be provided a $100 (US dollar) check in recognition of their time. To learn more and to reserve your spot, visit EB Health Research RSVP .
Patient Research – Testing of a Rare Disease Self-ID Tool
Global Genes has combined with Engage Health Inc. and LapidusData to develop a “Rare Disease Self- identification Tool” that will help patients and patient advocates understand that they may be affected by one of the 7,000 identified rare diseases. This tool will inform them that they are part of one of the largest disease communities in the world with many support resources available. The tool uses a set of questions that ask about common experiences of rare disease patients. If their answers indicate that they have a rare disease, the tool would then provide them with helpful resources that they may not have been aware of. The set of questions has been developed based on the experiences of rare disease patients, with input from members of the Global Genes Medical and Scientific Advisory Boards, and patient advocates.
We are still developing this tool, so our goal is to test whether the survey is asking questions that will identify people with rare diseases: do people with rare diseases give different answers from people without rare diseases? You can help by providing your own answers to the questions, which should take about 10 minutes. To access the survey, click on the this link.
Identification of Caregivers and Patients in OPMD
Our client would like to learn more about physicians’ experience with Oculopharyngeal Muscular Dystrophy (OPMD), including the number of patients who are diagnosed and how they are treated. If you are a physician who sees these patients and would like to participate in this brief survey please click here: survey. Your participation, which does not involve sharing confidential information, will help our client design future clinical trials.
Physician Experience in the MPS Disorders (MPS I Hurler and Hurler-Scheie and MPS II Hunter)
Our client, a leading biotechnology company focused on the development and commercialization of adeno-associated virus (AAV) gene therapy, is interested in learning more from physicians about their MPS I Hurler, Hurler-Scheie and MPS II Hunter patients that they see. By better understanding the experiences of healthcare providers, it will help them to increase the understanding of the diseases and their treatments and help understand if you are interested in clinical trials. Go to this link to participate.
Genetic Testing in Glucose Type-1 Deficiency Syndrome (Glut1 DS) – For Physicians
Our client is sponsoring a program for physicians offering a no-cost genetic testing for their patients with symptoms of Glut1 DS or a family history of the disease. The genetic test panel includes 3 key mutations which may account for up to 47% of paroxysmal dyskinesia cases, and takes between 2-4 weeks. If you are a physician who would like to participate in this free testing program, please click on this link to complete a brief survey and request the test kits.
Genetic Testing in GNE Myopathy – For Physicians
Our client is sponsoring a program for physicians offering a no-cost genetic testing for GNE Myopathy available for their patients with symptoms of GNE Myopathy or a family history of the disease. The free genetic testing program is for physicians who have patients in whom they suspect GNE Myopathy who have not undergone sequencing, or for those who have another diagnosis, but where GNE Myopathy is suspected (e.g. Limb Girdle Muscular Dystrophy). If you are a physician who would like to participate in this free testing program, please click on this link to complete a brief survey and request the test kits.
Physician Survey Regarding the Diagnosis and Treatment of Galactosialidosis and Sialidosis Type I
Our client would like to learn more about patients with galactosialidosis and sialidosis type I, including how they are diagnosed and treated. If you are a physician who sees these patients and would like to participate in this brief survey please click here: survey. Your participation, which does not involve sharing confidential information, will help our client design future clinical trials.