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Latest News: Treatments & Research

AveXis Community Update on Submitting their Application for AVXS-101

19 October 2018 / Posted in: Treatments & Research

AveXis yesterday submitted their regulatory applications for AVXS-101 in the U.S., Europe and Japan for use in infants with SMA Type 1. This is an important initial step toward the potential approval of AVXS-101, a gene replacement therapy candidate for the treatment of the underlying root cause of SMA.

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$3 million 'Breakthrough' prize for Developers Behind Spinraza

18 October 2018 / Posted in: Treatments & Research

An article in The Guardian, published yesterday, describes how C Frank Bennett, senior vice president of research at Ionis Pharmaceuticals, and Adrian Krainer of Cold Spring Harbor Laboratory, have won the prize for developing Spinraza.

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Have you visited an NHS ‘genetics service’ or ‘genomic medicine service’ in the last two years?

18 October 2018 / Posted in: Treatments & Research

Researchers are conducting a study to explore people’s views about how patient data could be used as part of future genomic medicine services in the NHS.

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Biogen Response to the Charities' August 28th Joint Letter

09 October 2018 / Posted in: Treatments & Research

In our letter, we urged both Biogen and NHS England to be as flexible as possible in their discussion on the price of nusinersen. Read their reply here.

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Consultation About Spinal Muscular Atrophy UK's Priorities

09 October 2018 / Posted in: Information, Treatments & Research, Support

We are now starting to work out priorities for the new organisation, including how and when the funds we hope to raise should be spent. We are very keen to hear the views of as many people in the SMA Community as possible. The survey closes on 11th November.

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Biogen’s ‘Biomarkers’ Research to Find Ways to Tell Whether a Medication Has Been Successful

09 October 2018 / Posted in: Treatments & Research

At the 23rd International Annual Congress of the World Muscle Society, pharmaceutical company Biogen presented the findings of blood tests done on over 300 participants receiving nusinersen in their clinical trials.

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New Results from Biogen’s NURTURE Study of Pre-Symptomatic Infants with SMA

09 October 2018 / Posted in: Treatments & Research

At the 23rd International Annual Congress of the World Muscle Society, pharmaceutical company Biogen announced its latest findings.

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Scottish Government Announces New Pathway Opens for Ultra-Orphan Treatments

09 October 2018 / Posted in: Treatments & Research

Yesterday’s announcement gives hope that nusinersen could soon be reassessed for access for those with SMA Type 2 or 3 in Scotland.

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Roche Announces Some Results from their Firefish and Sunfish Trials

09 October 2018 / Posted in: Treatments & Research

On 3rd October at the 23rd International Annual Congress of the World Muscle Society, Roche presented some of the results of their clinical trials of the treatment risdiplam.

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Campaign to Protect ERNs - Add Your Voice

08 October 2018 / Posted in: Treatments & Research

Genetic Alliance UK has launched a campaign to ensure the UK's continued participation in European Reference Networks (ERNs) following our exit from the European Union.

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