Virtual Support Group for Male Patients and Caregivers

Recently I met a lovely man named Dave Ross who has a rare disease called Cowden Syndrome. 2 years ago he set up a virtual support group for male patients and caregivers to help support the mental health side of living with a Rare Disease/Disorder. David has just set up additional meetings for...

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If Not Me, Who?

KEY POINTS In new research, adults with rare disorders discussed their key challenges and how they are combatting them. Lack of awareness among healthcare providers and the public leads to poor medical care and stigma. Many people with rare diseases take it upon themselves to engage in...

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Nexviazyme is under review by Medsafe NZ

Nexviazyme is a 2nd generation Enzyme Replacement Therapy (ERT) for Pompe disease.  Genzyme Corporation has put an application in to Medsafe NZ for assessment and approval. Then we need to cross our fingers that PHARMAC will approve it for funding! Nexviazyme status at Medsafe Information on...

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2022 RARE Patient Advocacy Summit

Rare Life. Bonded Together September 12-14, 2022 San Diego, CA The first conference attended since WORLD in March 2020!  It was great to be able to get out amongst people again. It was a full schedule with many topics discussed.  I was inspired by all of those who strive to keep showing up,...

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