The COVID-19 pandemic represents an enormous world-wide challenge both medically and economically. This challenge is further intensified multiple fold for the rare disease population including lysosomal storage disease (LSD) patients and families.  Patients with LSDs and other rare conditions are already regarded as the “fringe”, and their medical care is not seen by many in the establishment as a primary concern during such large scale public health disasters. However, rare disease patients of all ages, belong to the most vulnerable population not only because of their susceptibility to the catastrophic complications of the COVID-19 infection, but also their need of continued access to high level medical care, life saving but expensive therapeutics and/or participation to ongoing clinical trials, all of which are hampered during the COVID-19 outbreak. These patients are usually cared at highly specialized academic centers in major cities, currently the epicenters of the outbreak.

While social distancing and stay-at-home orders have been issued across various countries as a method to slow the curve of COVID-19 infections, it presents the rare disease community with its own set of unique complications. Enzyme replacement therapy (ERT) is one of the most accepted forms of treatment for various LSDs, but ERT requires periodic visits to hospitals or infusion clinics for treatment and medical evaluation by their care providers. While most of the medical practices switch to telemedicine, this is simply not an option for these patients.

In addition to the clinical management issues, it has to be noted that patients with LSDs are more susceptible to infections and complications from a viral infection due to their inherent immune-compromised states. Moreover, patients with LSDs also suffer from multiple morbid conditions, such as lung disease, kidney failure, cardiomyopathy, osteonecrosis, respiratory muscle weakness, and history of stroke. Some of the pediatric patients have neurodegenerative disorders as well.

GRIDS2020-Virtual summit will focus on the unique challenges in the time of COVID-19 pandemic that are faced by medical community as well as the LSD patients and families. GRIDS2020-Virtual Summit will provide a platform for physicians, researchers and other health care providers from across the globe to share their distinct experiences and hardships in management of rare diseases. These shared experiences and lessons learnt would serve as a way to establish much needed guidelines to be better prepared for large scale global events long after the COVID-19 pandemic has passed.

Global learning objectives

• 1. Adaptation and continuity of care for lysosomal Storage disorders during COVID-19 pandemic

• Get introduced to need for continuity of care for lysosomal storage disorders during COVID-19

• Discuss the global impact of COVID-19 on lysosomal storage disorders

• Learn about effect of COVID-19 on different treatment strategies for lysosomal diseases

• Gain knowledge about management of enzyme replacement therapies in COVID-19 high-risk patient population

• Discuss how to balance social distancing and care continuum

  2. SARS-CoV-2 infections and pathogenic pathways in lysosomal storage disorders      

• Gain knowledge about the impact of COVID-19 on pathogenic pathways inherent to lysosomal storage disorders

• Review basic mechanisms of coronavirus infections, and discuss mechanistic pathways relevant in lysosomal disorders

• Learn about targeting autophagic and endo-lysosomal dysfunctions inherent to LSDs

• Gain insights into the immunopathogenesis in LSDs and whether it plays a role in COVID-19 susceptibility and severity

• Discuss about increased vulnerability of multi-systemic disorder patients to COVID-19

  3. Long-term impact of COVID-19 pandemic on management and treatment of rare diseases

• Learn about long-term Impact of COVID-19 and Lessons learnt to prepare for pandemics

• Discuss about principles of development of vaccines and other specific therapies for COVID-19 infections, and whether there are any specific concerns for  LSD patient population

• Review inpatient management of COVID-19 patients and discuss specific recommendations for patients with LSDs (such as patients with Fabry disease and cardiomyopathy or patients with Pompe disease and respiratory muscle involvement)

• Gain insights into the unique challenges faced by rare disease community in the face of world-wide pandemic

• Discuss about compiling the ‘Lessons learnt’ register from COVID-19 experience for future preparedness

• Learn about relevance of collaborations and partnerships to overcome the pandemic challenges