Patient Experience Survey 2020
This survey by Genetic Alliance is designed to capture the experience of patients affected by rare and undiagnosed conditions, and their carers. The survey should take approximately 30 minutes to complete.
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This survey by Genetic Alliance is designed to capture the experience of patients affected by rare and undiagnosed conditions, and their carers. The survey should take approximately 30 minutes to complete.
Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in RNA-targeted therapeutics, announced today that the European Medicines Agency (EMA) has granted orphan drug designation to ION373 for the treatment of people with Alexander disease, a severe, progressive and debilitating condition that can eventually result in death due to loss of control over autonomic functions like breathing. Infants and young children who develop the condition rarely survive beyond their teenage years or young adulthood.
The BIMDG psychologists group work with individuals and families affected by inherited metabolic disorders (IMD).
