Patients and science are at the center<br />of everything we do

Our Stories
  • Photo from Rare Disease Day video
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    Make Your Pledge4Rare this Rare Disease Day
    RARE DISEASE
  • Image from the Medikidz comic book
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    Medikidz Cartoon Help Kids Understand Late-Onset Pompe Disease
    POMPE DISEASE
  • Photo of Brad, Cold Agglutinin Disease patient
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    Brad’s Story: Living with Cold Agglutinin Disease
    RARE BLOOD DISORDERS
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Research Pipeline

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Patient Access to Treatment

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50 Binney Street
Cambridge, MA 02142
Tel: 617.252.7500 | Toll-free: 800.745.4447
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