2022 RARE Drug Development Symposium

The Rare Drug Development Symposium, in partnership with the Orphan Disease Center of the University of Pennsylvania, brings together rare disease advocacy leaders and researchers to identify barriers to research, envision solutions, and accelerate progress for our communities. This two-day, in-person-only summit offers a unique opportunity to engage in interactive small-group workshops with experts and …

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11th European Conference on Rare Diseases and Orphan Products

Virtual

The European Conference on Rare Diseases and Orphan Products is recognised globally as the largest, patient-led rare disease event in which collaborative dialogue, learning and conversation takes place, forming the groundwork to shape future rare disease policies. Please contact martina.bergna@eurordis.org for more information.

Webinar: Importance of autopsies in leukodystrophies’​ by Marianna Bugiani

Virtual

Educational webinars on RARE neurological, neuromuscular and movement disorders jointly organized by the European Reference Networks for Rare Neurological Diseases (ERN-RND) and Neuromuscular Diseases (ERN EuroNMD) and the European Academy of Neurology (EAN). The speaker is Neuropathologist at the Amsterdam University Medical Center, the Netherlands. Time Jul 12, 2022 03:00 PM in Amsterdam, Berlin, Rome, …

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Hunter’s Hope 2022 Medical and Family Symposium

Holiday Valley Resort – Ellicottville, NY

The Hunter’s Hope Medical and Family Symposium is a time for Leukodystrophy families, medical providers, newborn screening lab personnel, industry partners, and researchers from around the world to gather for learning, sharing, and Hope.

Leukodystrophy Awareness Month

September is Leukodystrophy Awareness Month!  A leukodystrophy is a type of disease that causes degeneration of the myelin, or "white matter" of the brain.  Every individual requires healthy production of myelin for speed and accuracy of nerve impulses within the brain.  Without a healthy myelin sheath, your nerves can’t send and receive signals properly.

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