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January 2019:

On 25 January 2019, the inaugural ATTR Patient and Family Info Day was held at the Royal Free Hospital. The day was well attended: The Atrium conference venue at the Royal Free was filled to capacity by 130 attendees – patients and their relatives and friends. The day was organised by the UK ATTR Amyloidosis Patients’ Association (UKATPA). The morning involved some formal talks – speakers included Profs Hawkins and Gillmore from the NAC and Prof Reilly from the National Hospital for Neurology and Neurosurgery in London. Break-out sessions in the afternoon focussed on particular relevant topics and enabled discussion and sharing of information in smaller groups. The day was managed by NAC staff volunteers. Here are some videos from the day: 

A promotional / montage video of the day

An interview with David and Sue Gregory

Professor Hawkins’ presentation from 25 Jan

Professor Gilmore’s presentation from 25 Jan

Dr Lane’s presentation from 25 Jan 

Carlos Heras-Palou presentation from 25 Jan

Local press article about the infoday

December 2018:

The NICE assessments of Patisiran and Inotersen have been made public on their website. These recommendations are not final guidance and may change after consultation.  The UK ATTR Amyloidosis Patients Association is involved in the NICE process. Our summary of the significance of these assessments is as follows:

NICE assessment of Patisiran.

The National Institute for Health and Care Excellence (NICE) has published an Evaluation Consultation Document (ECD) on its assessment of patisiran. It states that “Patisiran reduces disability, increases quality of life and is innovative”. However, there remain some questions about its cost-effectiveness, therefore NICE state that “Patisiran is not recommended, within its marketing authorisation, for treating hereditary transthyretin-related amyloidosis in adults”.
It is important to keep in mind that this is an initial draft guidance and further discussions regarding the cost effectiveness of the drug will be held. UK ATTR Patient Association expects that NICE, Alnylam and NHS England will continue to work towards making patisiran available to patients in England and Wales. A final decision is anticipated in the new year.
Patisiran (Onpattro®) is a ribonucleic acid interference agent that suppresses transthyretin (TTR) production by the liver (including abnormal TTR). It is administered once every 3 weeks by intravenous infusion at a dose of 0.3 mg/kg. It has a marketing authorisation in the UK for treating ‘hereditary transthyretin-mediated amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy’.

NICE assessment of Inotersen.

The National Institute for Health and Care Excellence (NICE) has published an Evaluation Consultation Document (ECD) on its assessment of Inotersen. It states that “Inotersen slows progression of the disease considerably and is innovative”. However, there remain some questions about its cost-effectiveness, therefore NICE state that “Inotersen is not recommended, within its marketing authorisation, for treating polyneuropathy in adults with hereditary transthyretin-related amyloidosis”.
It is important to keep in mind that this is an initial draft guidance and further discussions regarding the cost effectiveness of the drug will be held. UK ATTR Patient Association expects that NICE, Akcea and NHS England will continue to work towards making Inotersen available to patients in England and Wales. A final decision is anticipated in the new year.
Inotersen (Tegsedi, Akcea Therapeutics) is a novel, first-in-class 2’-O-2- methoxyethyl phosphorothioate antisense oligonucleotide that inhibits production of transthyretin (TTR) in adults with hereditary transthyretinrelated (hATTR) amyloidosis. Inotersen has a marketing authorisation for the treatment of stage 1 or stage 2 polyneuropathy in adults with hATTR amyloidosis

September 2018:

Recent press coverage of ATTR amyloidosis and treatment advances:

– BBC News
– iNews

The National Amyloidosis Centre is holding an ATTR Amyloidosis Patient and Family Infoday at the Royal Free Hospital on Friday 25 January 2019. The Infoday will be held in the Royal Free Atrium 10am-3pm. See flyer below for more details:

ATTR InfoDay Advert