International Events

For the first time, an international conference will bring together liver and muscle types of glycogen storage disease (GSD). World experts will discuss the state of research and therapies for the muscle and liver types in parallel sessions. The exchange of experiences will be stimulated by plenary sessions, addressing the challenges of rare diseases, developing therapies for rare diseases, and the benefits of building communities.

Patient group representatives will complete the picture by contributing the patients' perspective. Special sessions for the most rare GSD types are an option, as well as workshops for specific topics. Scientists can expect a high-level scientific conference; patients can use it as a forum for first-hand information and community. The conference will contribute to a better understanding of the GSDs, encourage progress in therapies and disease management, and facilitate the formation of networks of both scientists and patients.

The conference program will cover the GSD liver types I/III (von Gierke/Cori disease) and the GSD muscle types II/V (Pompe/McArdle disease) as well as other rare GSD types.

The conference language is English.

International MPS Network Meeting at Rinku Osaka Japan has ended today. From this meeting we can see advance country which have a establish SYSTEM will take full care of their patient benefit. The patients are either protected under Orphan Drug Act(Taiwan, Japan, US) or via law suits against the federal government like in Brazil and some countries in South of America. 

LSD patients need Enzyme Replacement Therapy (ERT) which cost average RM 1 mil per patient per year. MLDA is stuck now as there’re still 13 patients awaiting for ERT. We do not have an Act here, and we do not even have a policy here, we only have a guideline to treat, so a guideline will always remain as a guideline. Budget challenge kill all these patients, it’s real sad to say this but this is the fact. 
What you can do to assist MLDA is to help us get more people to LIKE our page so that our voice could be heard by our Prime Minister.

MLDA had participated in the World Orphan Drug Congress Asia 2013 which has held in Singapore recently.

World Orphan Drug Congress Asia 2013 is Asia’s most strategic orphan drug industry event. This event is dedicated to fostering partnerships and collaboration amongst industry, governments, and patients.
Bringing together more than 150 orphan drugs stakeholders, this event will focus on expediting orphan drug development and articulating its value, from discovery to clinical development, to licensure, to manufacturing, to launch, and to sustainability
of supply, so that manufacturers are guaranteed full and speedy reimbursements. This event will also cover a range of topics that are pertaining to market access and patient care.

MLDA will attend 2013 International MPS Meeting which is going to held in Osaka Japan this year from 1-3 Aug 2013.

Venue:  World Wide

Rare Disease Day is an annual, awareness-raising event co-ordinated by EURORDIS at the international level and the National Alliances of Patient Organisations at the national level

February 29, 2012 marks the fifth international Rare Disease Day coordinated by EURORDIS and organised with rare disease national alliances in 25 European countries.On this day hundreds of patient organisations from more than 40 countries worldwide are organsing awareness-raising activities converging around the slogan “Rare but strong together”.

For more information, please visit our website at www.rarediseaseday.org

  1. The 3rd Educational Forum for Families and Caregivers on MPS by Taiwan MPS Society
  2. RDSS Piano Recital Charity Fundraiser