Rare Diseases
Science in Rare Diseases
Rare diseases are more common than most people imagine
Rare Disease Day raises awareness for the 300 million people worldwide living with a rare disease, along with their families and carers. Over 6,000 rare diseases are characterized by a wide range of conditions and symptoms that can vary not only between diseases but also among individuals with the same condition. Take the opportunity to support equitable access to diagnosis, treatment, healthcare, social support for those affected by rare diseases.
With Pompe Disease : Time is muscle LOPD Treatment Guide for appropriate and timely intervention
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