A man living with a rare and degenerative disorder is making a heartfelt plea to officials to get approved for a treatment that will help to prevent further damage to his lungs and liver. Alpha-1 Antitrypsin Deficiency is a disorder caused by a missing blood enzyme that leads to liver
HMR have opened up their trial to now accept people who are taking inhalers. If you are PiMZ, PiSZ, or PiZZ, you might be able to join in this new exiting trial. Please contact HMR direct using the contact email shown below quoting 20B005 in your email. If you have
RCPCH &Us an @BPSUtweet have a survey for children & young people aged 13-25, who have a rare disease & live in the UK. We want to hear your views. To complete the survey visit: https://tinyurl.com/xmadvv8x #voicematters #youngpeoplematter
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