The Second Joint International Symposium on Neuroacanthocytosis (NA) and Neurodegeneration with Brain Iron Accumulation (NBIA) will highlight new breakthroughs in NA/NBIA – towards unraveling underlying mechanisms. Programme to include: autophagy; clinical studies; erythropoiesis; new genes; pathophysiology; protein-misfolding; treatment strategies / round table discussions with patient organizations and basic and clinical...
Events
Innovative Approaches for Addressing the Needs of Ultra Orphan Patient Populations Real Solutions for Today’s Challenges There is a new model for pharma to consider for engaging with patients safely. It puts a greater focus on patient advocacy while identifying the expectations and needs of rare disorder patients and caregivers....
The 2nd Annual Orphan Drugs Summit 2012 is the platform for experience sharing amongst Orphan Drugs professionals, looking at alternative ways to raise funding, to overcome development challenges and to partner with stakeholders to ensure orphan drugs reach the market of rare diseases patients. To download this year’s agenda, please...
The 19th Annual Chromosome 18 Registry Family Conference will be held in the home city of The Registry, beautiful San Antonio, Texas. Our conference will be held downtown at The Riverwalk Marriott, situated on the famous San Antonio Riverwalk. The conference will kick off on Sunday night, July 29th, with...
The International Workshop “Rare Diseases and Orphan Drug Registries” is organized in the framework of the EPIRARE project (“European Platform for Rare Disease Registries”, co-funded by EU Commission, DG SANCO). It is open to scientists, clinicians, patients’ and parents’ Associations, policy makers and enterprises. The main aims of the workshop...
The First European Days of Albinism will take place in Paris on Saturday 27th and Sunday 28th October 2012. This conference has received the support of the European Society for Pigment Cell Research and of Alliance Maladies-Rares. This conference will gather representatives of European organizations of people with albinism, doctors...
Co-funding Institute(s): Welcome to The Tenth International Catecholamine Symposium (XICS) co-funded by the National Institute of Child Health and Human Development (NICHD) and the Office of Rare Diseases Research (ORDR) Venue Ailomar Conference Grounds, Pacific Grove, CA, United States Description The Tenth International Catecholamine Symposium (XICS) will emphasize integration of...
Welcome to the Outcome Measures and Infrastructure for Phase III Studies in Batten Disease, JNCL co-funded by the National Institute of Neurological Disorders and Stroke (NINDS) and the Office of Rare Diseases Research (ORDR) Description The main objective of the meeting is to bring together clinical research experts in JNCL...
Welcome to the RDCRN 3rd Conference on Clinical Research for Rare Diseases co-funded by the National Institute of Neurological Disorders and Stroke (NINDS) and the Office of Rare Diseases Research (ORDR) Description Clinical research in rare diseases presents a number of challenges and unique issues that are not usually considered...
Welcome to the Fourth International MHE Research Conference co-funded by the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) and the Office of Rare Diseases Research (ORDR) Description The goals of the conference are: to provide a forum to share the most recent and unpublished findings in these...
Green fees $100 per golfer, includes cart, lunch, refreshments and dinner. Fundraising event in honor of Landon Hartz. Proceeds to benefit the Cystinosis Research Foundation.
Sunrise Mall Kitchener ( home depot parking lot ) Charity BBQ support Cystinosis ( Gabbies Wish ) 50/50 draw raffle table Sound off Competition Club tug of war… DJ ( all day music ) Glass trophies for 1st and second in class eveyone is welcomed db drags…bass race SPONSORS: Bast...
We invite you to attend our 31st annual National Family Conference in magical Orlando, Florida. These four days will change your life. We have hosted this conference for three decades for individuals who are affected by ectodermal dysplasias and their loved ones. You will receive the latest updates on research...
The Human Genetics Society of Australasia is proud to invite you to attend the 36th Annual Scientific Meeting in Canberra, Australia from Sunday 22 to Wednesday 25 July 2012. Who should attend? This meeting will have broad appeal for all scientists, clinicians and allied health professionals with an interest in...
This conference aims to provide an exchange of ideas about genetics, genomics and systems biology in the context of vascular biology to increase our understanding of vascular disease. As such, this innovative workshop will be of interest to a broad spectrum of investigators that do not normally interact with one...
3rd Pan-European Conference on Haemglobinopathies and Rare Anaemias, 25-26 October 2012, Cyprus EPHA member the Thalassaemia International Federation has announced the organisation of the 3rd Pan-European Conference entitled ‘Haemglobinopathies and Rare Anaemias: Towards the Future’. The event will be held under the auspices of the Cyprus Presidency and in close...
The call to treat rare diseases has become louder around the world in recent years than ever before. Supportive organisations including patient groups, national health authorities, health trusts, academia, HTAs, pharmaceutical and biotechnical companies are now progressively growing in number and sophistication in their approaches to medicinal care for those...
Welcome to the WFH 2012 World Congress! The WFH World Congress is the single largest event in the WFH calendar, and is very important to the global bleeding disorders community. Every second year doctors, scientists, healthcare workers, people with bleeding disorders and hemophilia organizations gather to learn about the latest...
Expedition Mt Kinabalu Calling all adventurers! Lace up your hiking boots! Coalition Duchenne will hold its second annual Expedition Mt Kinabalu, Summit of Borneo, on August 25th, 2012. Join this exciting global event and be one of 60 international climbers to raise awareness and funding to find a cure for Duchenne...
Join a host of health care professionals and paediatric experts from around the globe as we share, explore and discuss the latest developments in paediatrics and how they can be implemented into your daily life to improve the way you practice. Now in it’s fourth year, Excellence in Paediatrics provides...