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email forum - by joining the group e-mail forum you will be in contact with Alpha patients, their families, carers and friends living throughout the UK and overseas. We are a friendly group, and between us we have a wealth of knowledge and experience to share. There is always someone to help with any worries or queries you might have.

What is Alpha-1 Antitrypsin Deficiency?

Alpha-1 Antitrypsin Deficiency also known as Alpha-1, A1AD or AATD is an inherited, genetic condition that is passed on from generation to generation. As the name suggests it is a deficiency of alpha-1 antitrypsin (AAT) in the bloodstream. AAT is an enzyme produced in the liver to help protect the tissues of the body during infections. The low level of AAT in the blood occurs because the AAT is abnormal and cannot be released from the liver at the normal rate. This leads to a build up of abnormal AAT in the liver that can cause liver disease and a decrease of AAT in the blood that can lead to lung disease.

Top Story

One step closer to the first UK drug license for augmentation therapy for patients with severe alpha-1 antitrypsin deficiency

CSL Behring has announced today that the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended granting a market authorisation to CSL’s alpha-1 antitrypsin augmentation therapy product Respreeza®.

Receiving a positive CHMP opinion (See EMA Website) marks an important milestone in the process of Respreeza® obtaining a market authorisation (= drug license) in all EU Member States, including the UK. The CHMP positive opinion will be transmitted to the European Commission (EC) to start the EC decision-making process on granting a centralised European drug license for Respreeza®, which we hope to be concluded later this year.

Lutz Bonacker, Senior Vice President & General Manager Commercial Operations, Europe, CSL Behring said: “CSL Behring continues to make strides towards fulfilling our promise to improve the lives of our patients. This positive opinion from CHMP brings us closer to providing Respreeza® as a new treatment option to the AATD community in Europe.”

However, once a drug license is granted by the EC, access to Respreeza® to UK patients will be subject to reimbursement of the drug by the NHS. As use of Respreeza® will be indicated in adults with documented severe alpha-1 antitrypsin deficiency and evidence of progressive lung disease as evaluated by a healthcare professional experienced in the treatment of Alpha-1, it is important that patients gain access to the care provided by a physician with sufficient experience in the field of Alpha-1, e.g. at the recently established West Midlands Alpha-1 Services
Alpha-1 UK Support Group
14th Annual Social Gathering/Information Day

Bentley Hotel Leisure Club and Spa Lincoln
Saturday 12th September 2015


Due to popular demand this year’s Alpha-1 UK Support Group 14th Annual Social Gathering will again be in the historic city of Lincoln on Saturday 12th September 2015 at the Bentley Hotel Leisure Club and Spa. The event is one of the largest gatherings of Alphas and their families in the UK where you can meet and socialise with fellow Alphas and be updated
by leading experts in the field of Alpha-1 Antitrypsin Deficiency.



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Latest News

Step Closer to First UK Drug License Augmentation ...
29 Jun 2015 16:37

One step closer to the first UK drug license for augmentation therapy for patients with severe alpha-1 antitrypsin deficiency CSL Behring has annou [ ... ]

Read More
Trial Results Alpha-1 Augmentation Therapy
28 May 2015 18:20

The Lancet publishes RAPID trial results showing effectiveness of Alpha-1 augmentation therapy. The Alpha-1 Foundation on Thursday congratulated the  [ ... ]

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New Specialist NHS Clinics for Alpha-1
28 Apr 2015 17:48

New NHS Funded Multi Disciplinary Service for Alpha-1 Patients

Specialist NHS clinics for patients with alpha 1-antitrypsin deficiency will be running  [ ... ]

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Clinical Trials Begin to Treat Alpha-1 Liver Disea...
25 Feb 2015 14:29Clinical Trials Begin to Treat Alpha-1 Liver Disease

TAP collaborator Arrowhead begins clinical trials on ARC-AAT to treat Alpha-1 liver disease. A groundbreaking trial of a potential treatment for Alpha [ ... ]

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