Cure Cystinosis International Registry
Monday 30th of August 2010
Dear Cystinosis Community Families and Friends:
We are excited to announce that the Cure Cystinosis International Registry (CCIR) has been launched! This official international cystinosis registry should not to be confused with a recently conducted phone survey or any other web survey about cystinosis.
The CCIR is the only registry created specifically for the entire global cystinosis community. It was created by medical and scientific cystinosis experts with leadership and input from many international cystinosis organizations and cystinosis family foundations. The CCIR is the only secure registry with a professional curator, which ensures complete patient privacy, as well as maximum scientific effectiveness.
The sole purpose of CCIR is to connect those with cystinosis to the research community whose goal is to learn more about cystinosis and ultimately to find a cure for cystinosis. The Cystinosis Research Foundation (CRF), in collaboration with the Cystinosis Foundation (CF) and many other cystinosis leaders and organizations from around the world, worked to create a patient-based international cystinosis registry.
In order to successfully accelerate and maximize the research process, we need participation from every person/family with cystinosis. We urge you to register today ˆ and then to spread the word to others who have cystinosis. With so many exciting research discoveries being made and so many more on the horizon, timing is crucial and participation is essential. You can register now at www.cystinosisregistry.org.
Our goal is to achieve 100 percent participation from the cystinosis community so we can provide cystinosis researchers who are committed to finding better treatments and a cure with the most accurate and complete data in the world. All personal information provided to CCIR will be maintained in a secure database accessible only by the CCIR Curator, Ms. Betty Cabrera at the University of California, San Diego. Information that could identify participants and their family members will not be shared without their expressed approval. The registry questionnaire is comprehensive, covering areas from diagnosis, medications, transplants to quality-of-life questions. Upon completion, registrants will be able to compare their responses to others in the cystinosis community, thus providing them a glimpse of how they fit into the larger cystinosis community.
We have included brochures we hope you will share with other cystinosis families, as well as with the doctors and others who provide your healthcare services. The registry is web-based so you will need to register online, but you may also request a hard copy of the registry questionnaire from the Cystinosis Research Foundation. Contact information is listed on the CCIR brochure.
We encourage you to use chat rooms, family forums, Twitter and Facebook to help spread the word! Participation in the registry offers every member of our community a role in the quest for a cure for cystinosis. Please join the cystinosis researchers, clinicians, scientists and cystinosis family members and foundations from around the world and register with CCIR ˆ be part of the cure!
Best regards,
Nancy Stack, President Cystinosis Research Foundation
We are excited to announce that the Cure Cystinosis International Registry (CCIR) has been launched! This official international cystinosis registry should not to be confused with a recently conducted phone survey or any other web survey about cystinosis.
The CCIR is the only registry created specifically for the entire global cystinosis community. It was created by medical and scientific cystinosis experts with leadership and input from many international cystinosis organizations and cystinosis family foundations. The CCIR is the only secure registry with a professional curator, which ensures complete patient privacy, as well as maximum scientific effectiveness.
The sole purpose of CCIR is to connect those with cystinosis to the research community whose goal is to learn more about cystinosis and ultimately to find a cure for cystinosis. The Cystinosis Research Foundation (CRF), in collaboration with the Cystinosis Foundation (CF) and many other cystinosis leaders and organizations from around the world, worked to create a patient-based international cystinosis registry.
In order to successfully accelerate and maximize the research process, we need participation from every person/family with cystinosis. We urge you to register today ˆ and then to spread the word to others who have cystinosis. With so many exciting research discoveries being made and so many more on the horizon, timing is crucial and participation is essential. You can register now at www.cystinosisregistry.org.
Our goal is to achieve 100 percent participation from the cystinosis community so we can provide cystinosis researchers who are committed to finding better treatments and a cure with the most accurate and complete data in the world. All personal information provided to CCIR will be maintained in a secure database accessible only by the CCIR Curator, Ms. Betty Cabrera at the University of California, San Diego. Information that could identify participants and their family members will not be shared without their expressed approval. The registry questionnaire is comprehensive, covering areas from diagnosis, medications, transplants to quality-of-life questions. Upon completion, registrants will be able to compare their responses to others in the cystinosis community, thus providing them a glimpse of how they fit into the larger cystinosis community.
We have included brochures we hope you will share with other cystinosis families, as well as with the doctors and others who provide your healthcare services. The registry is web-based so you will need to register online, but you may also request a hard copy of the registry questionnaire from the Cystinosis Research Foundation. Contact information is listed on the CCIR brochure.
We encourage you to use chat rooms, family forums, Twitter and Facebook to help spread the word! Participation in the registry offers every member of our community a role in the quest for a cure for cystinosis. Please join the cystinosis researchers, clinicians, scientists and cystinosis family members and foundations from around the world and register with CCIR ˆ be part of the cure!
Best regards,
Nancy Stack, President Cystinosis Research Foundation
News Letters
Sunday 14th of February 2010
Hi all,
If your looking for our past newsletters you can view in PDF format by clicking on the links below.
January 2003
June 2003
August 2004
January 2007
Apologies some of the photo's are missing from the January 2007 newsletter.
If your looking for our past newsletters you can view in PDF format by clicking on the links below.
January 2003
June 2003
August 2004
January 2007
Apologies some of the photo's are missing from the January 2007 newsletter.
DR Cysteamine Phase 2b Clinical Trial
Friday 29th of January 2010
Raptor Pharmaceutical Corp. today announced results from a Phase 2b clinical trial of its proprietary delayed-release cysteamine bitartrate in patients with nephropathic cystinosis. The trial, conducted at the University of California, San Diego General Clinical Research Center, evaluated safety, tolerability, pharmacokinetics and pharmacodynamics of a single dose of DR Cysteamine in nine cystinosis patients.
Phase 2b Clinical Trial Highlights:
DR Cysteamine demonstrated improved tolerability and the potential to reduce total daily dosage and administration frequency compared to immediate-release cysteamine bitartrate.
Pharmacokinetic evaluation showed that DR Cysteamine had a terminal half-life more than three times longer than the terminal half-life of immediate-release cysteamine bitartrate capsules.
Twice-daily DR Cysteamine may achieve the same pharmacodynamic result while using a daily dose 30% lower than immediate-release cysteamine bitartrate capsules administered four times daily.
No adverse events recorded during the clinical trial were determined by the principal investigator to be possibly or probably related to DR Cysteamine. Nine adverse events recorded in the clinical trial were determined to be possibly or probably related to immediate-release cysteamine bitartrate.
Raptor plans to meet with the Food and Drug Administration ("FDA") and European Medicines Agency ("EMEA") to discuss the results of its phase 2b study and its plans for a repeat-dose, pivotal, phase 3 clinical trial in cystinosis patients. Upon receiving FDA and EMEA concurrence, Raptor intends to initiate its phase 3 clinical trial at multiple sites in the US and Europe in the first quarter of 2010.
You can read additional information by clicking here
Phase 2b Clinical Trial Highlights:
DR Cysteamine demonstrated improved tolerability and the potential to reduce total daily dosage and administration frequency compared to immediate-release cysteamine bitartrate.
Pharmacokinetic evaluation showed that DR Cysteamine had a terminal half-life more than three times longer than the terminal half-life of immediate-release cysteamine bitartrate capsules.
Twice-daily DR Cysteamine may achieve the same pharmacodynamic result while using a daily dose 30% lower than immediate-release cysteamine bitartrate capsules administered four times daily.
No adverse events recorded during the clinical trial were determined by the principal investigator to be possibly or probably related to DR Cysteamine. Nine adverse events recorded in the clinical trial were determined to be possibly or probably related to immediate-release cysteamine bitartrate.
Raptor plans to meet with the Food and Drug Administration ("FDA") and European Medicines Agency ("EMEA") to discuss the results of its phase 2b study and its plans for a repeat-dose, pivotal, phase 3 clinical trial in cystinosis patients. Upon receiving FDA and EMEA concurrence, Raptor intends to initiate its phase 3 clinical trial at multiple sites in the US and Europe in the first quarter of 2010.
You can read additional information by clicking here
Next INTERNATIONAL cystinosis conference Italy Sept 2010
Friday 29th of January 2010
Hi everyone,
just letting you all know the next INTERNATIONAL cystinosis conference is being held in Italy Sept 2010
If anyone is even contemplating going to this conference please let me know so we can get an idea of how many from australia would like to go.
If possible please print out and give the flyers to your doctors when you next visit. If you haven't got a printer and would like me to send you copies just let me know.
It would be really nice if we could get some of our aussie doctors to attend. I have heard that Westmead will probably be sending a doctor Not sure which one yet. ( Jeff Fletcher NSW and Geoff Zhang NSW and Ian Hewitt in WA have already been to previous conferences)
Our Serena's doctor Dr Kulkarni said today that he might attend. They have almost a years notice. They can apply for funding for conferences. So lets get the info out there and you never know. It would be lovely to get a whole group from australia to go to the conference.
The Italians are trying to negotiate a good price for rooms and three meals each day . Will let you know info as it comes through.
But please let me know if you plan to try to get there.
Thinking of you all Sue Scott and family
just letting you all know the next INTERNATIONAL cystinosis conference is being held in Italy Sept 2010
If anyone is even contemplating going to this conference please let me know so we can get an idea of how many from australia would like to go.
If possible please print out and give the flyers to your doctors when you next visit. If you haven't got a printer and would like me to send you copies just let me know.
It would be really nice if we could get some of our aussie doctors to attend. I have heard that Westmead will probably be sending a doctor Not sure which one yet. ( Jeff Fletcher NSW and Geoff Zhang NSW and Ian Hewitt in WA have already been to previous conferences)
Our Serena's doctor Dr Kulkarni said today that he might attend. They have almost a years notice. They can apply for funding for conferences. So lets get the info out there and you never know. It would be lovely to get a whole group from australia to go to the conference.
The Italians are trying to negotiate a good price for rooms and three meals each day . Will let you know info as it comes through.
But please let me know if you plan to try to get there.
Thinking of you all Sue Scott and family