2nd Annual Syneos Health Rare Disease Day Symposium Despite significant progress fueled by technologies such as gene therapies, there are still no effective therapies for… Continue reading Syneos Health
Results for "light up for rare"
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When I was 8 years old I was diagnosed with a brain tumour at the back of my head. I was rushed to the hospital… Continue reading From Metal Head to Literal Metal Head
Founded in 1998, PTC is a global commercial, diversified biopharmaceutical company. At PTC, we are committed to a single purpose: Extending life’s moments for patients with… Continue reading PTC Therapeutics
How it all began My story began in 2008, only a few months after finishing one highschool year abroad. One day, it was a cold winter… Continue reading one half breath
Still not sure visual snow syndrome is a rare disease like people talk about. Feels like being near sighted and taking off your glasses. Like… Continue reading Visual Snow
Still not sure visual snow syndrome is a rare disease like people talk about. Feels like being near sighted and taking off your glasses. Like… Continue reading Visual Snow
Australian Sickle Cell Advocacy Inc (ASCA) is a not for profit organisation, a registered charity that provides support to Australians that are impacted by sickle… Continue reading Australian Sickle Cell Advocacy Inc
Swathi was born healthy on the 12th of April 2017. At the age of 6 months, upon prolonged boughts of pneumonia, a series of tests led to the shocking… Continue reading Malaysia Pompe Warrior
From Challenging Phases To Facing Challenges Story of Zoyeb from Chennai / India – ATAXIA Written by his father – Iftikhar ——————————————————————————————————————————- Zoyeb , who… Continue reading From Challenging Phases to Facing Challenges