Living with a rare disease brings numerous challenges. There are health impacts, psychosocial impacts, challenges related to delays in diagnosis, limits on access to treatment, and other issues that add to the burdens of patients and caregivers.
Listening to patients, families, advocates and healthcare providers reveals common themes of struggle and challenge, as well as inspiring stories of hope.
For its 2023 Rare Disease Day Event, Rocket Pharma brings together a diverse group of stakeholders to illuminate different dimensions of the rare disease experience.
• Meet a teenager with Pyruvate Kinase Deficiency (PKD) and his mother and hear about transitioning from pediatric to adult care with a rare disease.
• Meet another teenager and mother, both affected by Arrhythmogenic Cardiomyopathy (ACM), and hear how they deal with the risk of sudden cardiac death.
• Hear about the powerful work being done by patient advocacy groups to support the rare disease community, such as the SADS (Sudden Arrhythmia Death Syndromes) Foundation, PKD (Pyruvate Kinase Deficiency) Foundation, Thrive with Pyruvate Kinase Deficiency Organization, and Shaping Foundations.
Rocket invites all members of the rare disease community to join the live webcast of this event, which is free. Register through this form.
Learn more about Rocket Pharmaceuticals