Bioblast Pharma

Hope should not be rare

Patient advocacy

Patient Advocacy

Connecting patients with OPMD with the support they need

Patient Support

  • Living With OPMD »

    A dedicated patient-to-patient support community that is part of Ben’s Friends and provides patients and their families with a forum to share their thoughts and experiences with treatment and care.

  • Ben’s Friends »

    An organization that helps connect people with rare diseases and chronic conditions through a network of patient communities.

  • Genetics Home Reference (NIH) »

    An online resource that offers disease-related information to help patients and their families better understand the rare genetic disease that is affecting their lives.

  • Muscular Dystrophy Association »

    The world’s leading nonprofit health agency dedicated to finding treatments and cures for diseases such as muscular dystrophy and other neuromuscular diseases.

  • NORD (National Organization for Rare Disorders) »

    A national organization focused on helping patients with rare diseases and their families access relevant information, resources, medications, and support.

  • Muscular Dystrophy Campaign »

    An OPMD fact sheet that provides patient-friendly information about this rare genetic disease.

  • CORD (Canadian Organization of Rare Disorders) »

    Canada’s national network for organizations representing all those with rare disorders that advocates for improved health policy and healthcare systems for the rare disease community.

  • Muscular Dystrophy Canada »

    A national volunteer agency committed to eliminating neuromuscular disorders and alleviating the associated distress.

Learn about patients with SCA3