Rare diseases (RDs) affect over 300 million people worldwide. There are approximately 7,000 recognised RDs. RDs are usually genetic and are associated with severe debilitating symptoms which persist for a patient’s lifetime. RD patients face multiple challenges including delay in accurate diagnosis and significant unmet need for beneficial therapies. For 90 % of RDs there are no effective therapies. Medicinal Cannabis (MC) is used to relieve symptoms, such as pain, muscle spasticity and anxiety, which may be commonly experienced by patients with RDs. Being able to manage the symptoms and the progression of diseases, can positively impact patients, families and health care systems.peer-reviewe