Carbamoylphosphate synthetase deficiency
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Current topics:

• - What can we do to stay healthy while stuck at home?
  I feel discouraged about my weight loss.  The best thing for me is the pool, but they are all closed. 
• - US Approves First RNA Interference Drug
  Alnylam’s Onpattro (patisiran) can treat a rare hereditary disease by silencing a specific gene. The first therapy based on RNA interference (RNAi) gene silencing has been approved by the US Food a

Current topics:

• - What is the most frustrating or difficult aspect of living with a rare disease?
  Hello everyone,  How are you today? As you already know, a diagnosis of a rare disease can have a huge impact on one's life. It can bring new insights and information, but also many questions, cha
• - How were you diagnosed with a rare disease?
  Hello everyone, How are you doing? I thought I'd create this discussion to talk about being diagnosed with a rare disease! What were the first symptoms you experienced? How were you diagnosed? @A

Current topics:

• - COVID-19: How do you feel today?
  Hello everyone, I've opened this discussion so we can share our state of mind during this period of isolation! Let's come here everyday to share how we're feeling! What is the best way to stay pos
• - COVID-19 vaccine: What do you think?
  Hello everyone, How are you today? The pharmaceutical groups Pfizer (USA) and BioNTech (Germany), as well as Moderna (USA), announced last month that their vaccine candidates are around 94 and 95%
• - COVID-19: Should we fear a second wave?
  Hello everyone, How are you doing today? As countries throughout the world are and have been coming out of lockdown and life is begining to return to "normal", many people and health authorities ha