Registered non-profit  |  San Francisco, California, USA  |  info@iamgsd.org  |  © 2019 IAMGSD

CAMPAIGNS

Board members have established these campaigns on which to currently  concentrate our efforts and resources in order to achieve our objectives. Other work will continue, but these are our focus.
REDUCING THE AGE OF DIAGNOSIS

THE ISSUE

In McArdle disease, symptoms are frequently apparent before the age of 5 and certainly by age 10. Despite being taken to the family doctor at a young age, diagnosis is often delayed on average 20 to 25 years. The other muscle GSDs are mostly the same. Because undiagnosed people  are unable to understand how to manage their condition, many will damage themselves by doing the wrong activities or through inactivity. Most will also have significant psychological issues relating to self image, anxiety and depression.  

THE WORKPLAN

Our objective is to have every child diagnosed before the age of 10.  We will work towards this by:

  1. Raising awareness amongst Physical Education teachers.

  2. Helping to educate General Practitioners about muscle GSD.

  3. Making more child/parent centered diagnostic information available.

  4. Helping to educate pediatricians about muscle GSD.

PROMOTING STANDARDS OF CARE

THE ISSUE

At present, there are no internationally agreed Standards of Care for muscle GSDs. This presents a distinct challenge for both patients and clinicians. Inappropriate management can lead to poor patient outcomes and reduced quality of life. Given that the mainstay for managing muscle GSDs requires effective day-to-day self-management, standardization of care based on best practice is vital.  

THE WORKPLAN

Our objective is to see the dissemination of standards of care and best practice. We will work toward this objective by:

  1. Working with national GSD organizations, researchers and clinicians to inform the development of Standards of Care.

  2. Communicating the patient perspective to clinicians.

  3. Developing publications that are supported by current evidence-based research.

  4. Providing support/information to all clinicians who care for patients with muscle GSDs.

ENCOURAGING REGULAR PHYSICAL ACTIVITY

THE ISSUE

Individuals with McArdle disease experience varying degrees of symptoms, ranging from mild exercise intolerance to severely limited exercise intolerance with fixed muscle weakness. Physically active individuals are much more likely to improve their exercise intolerance and ultimately the clinical course of the disease. However, in order to achieve this, patients must have access to proper clinical management.  

THE WORKPLAN

Our objective is to raise awareness and provide support to patients and clinicians regarding the importance of regular physical activity (150 min/week). We will work toward this objective by:

  1. Disseminating research-based data on the benefits of regular exercise and exercise interventions.

  2. Providing practical resources that offer guidance on ‘how-to’ safely engage in exercise.

  3. Supporting clinicians who care for patients with muscle GSDs.

  4. Offering opportunities to ‘get moving’, such as the ‘Annual Walking Week’.

  5. Providing funding to researchers.

CONTRIBUTING TO RESEARCH

THE ISSUE

Clinical and epidemiological data on muscle GSDs is limited. With only a few clinicians worldwide seeing significant numbers of patients, research opportunities are limited. There is a distinct requirement for establishing networks of patients and their families on an international basis, and ensuring that patients’ needs are fed into the discussions on the research agenda.

THE WORKPLAN

Our objective is to contribute to the planning of research projects and to support and assist those projects. We will work toward this objective by:

  1. Collaborating with researchers on translational research projects. 

  2. Promoting patient involvement in the Euromac Registry.

  3. Participating in committees that are responsible for research decision-making.

  4. Disseminating information about research to patients and family members. 

  5. Providing funding to researchers.