PKD PATIENTS FORUM – April 3 rd, 2017 at Hammersmith Hospital, London.
On april 3rd there will be a patient engagement forum in London to inform us (patients and relatives) and the physicians about the gene therapy clinical trial for PKD called “ForGetPKD” and to start contacts for potential future enrollment.
During this event we will get more information about gene therapy and how it’s used to treat thalassemia and sickle cell disease and how it will be tested to treat PKD.
You can attend the event for free but have to register by contacting Nicola Window: firstname.lastname@example.org.
More info: PKD Forum Announcement
What if you’re young and because of your Pyruvate Kinase Deficiency your Ferritin is too high. In some cases, when other medicines don’t work or are not efficient, the doctors advice to use Desferal. But that’s not an easy thing to do and especially when not you’re a young boy. Using Desferal involves needles and a pump that has to stay several hours and sometimes multiple times a week.
A Very Brave young man with Pyruvate Kinase Deficiency made a video together with his mother in where he explains how it works. Hos goal is to teach people all about it and he has done a great job explaining it, without making it scary.
So we are Very proud to share this video and we hope that it will help other people that have to use Desferal.
21 and 22th of November 2015 was a special weekend for people with Pyruvate Kinase Deficiëncy. For the first time there was a symposium on Rare Anaemias that had a focus on Pyruvate Kinase Deficiëncy. For this occasion there where allot of people with this disease invited to participate.
Professionals from around Europe and America where invited to share the developments that are now in progress, such as new treatments, new learned knowledge about the illness and its side-effects and the care for patiënts in the hospital. Beside all the professionals Maaike Eijgenraam was invited to tell the professionals the perspective of the patiënts.
All in all the conference was place buzzing with new ideas and it was clear that the interest of professionals (doctors, pharmaceutical industries and scientist) where more and more focussed on PKD.
The presentation can now be seen online.
Written presentations and summaries :
Agios Pharmaceuticals continues to enroll PKD patients in their Patient Interview Study to learn more about living with PK deficiency. The interviews take about 1 hour to complete. If you would like more information about this work, please click here to read the flyer.
Interviews are currently being conducted for patients and caregivers living in the US. Plans are underway to include patients and caregivers living in Canada and the EU. More information will be provided once this project is approved in these locations.
Agios is developing a questionnaire that will help them to understand the impact of PK deficiency on both patients and caregivers. To learn this information, a trained interviewer will be conducting face to face interviews with adults, adolescents, and young children with PK deficiency, and caregivers of children with PK deficiency. Continue reading Patient Interview Study
The 6th European Symposium on Rare Anaemias is quickly approaching. Organized by the European Network for Rare and Congenital Anaemias, this first Dutch-Belgium meeting for patients and health professionals will take place November 21-22, 2015 in Amsterdam, The Netherlands. This event is of no cost to patients and their families and will be presented in Dutch and English. Continue reading European Symposium on Rare Anaemias
Agios has launched a new website called Drive PK trial, which offers patients, caregivers, and healthcare professionals information about their phase 2 clinical trial for the oral medication, AG-348. This medicine has been developed as a potential treatment for pyruvate kinase deficiency. Due to a deficiency in the enzyme, pyruvate kinase, people with pk deficiency are anemic, which means they have a reduced number of healthy red blood cells. The study is being conducted to determine Continue reading Agios launches the DRIVE PK Trial website