New clinical research study for Fabry from Protalix

The BALANCE Study is a clinical research study that will compare the safety and efficacy of an investigational medication (a potential new medication) with an approved medication (agalsidase beta, also known as Fabrazyme) in patients with Fabry disease. The investigational medication is a type of medication called “enzyme replacement therapy,” or ERT. The investigational medication … Continue reading “New clinical research study for Fabry from Protalix”

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The SMC decision regarding Migalastat (Galafold)

The SMC decision regarding Migalastat (Galafold) was announced on 7 November 2016.  The relevant Detailed Advice Document can be found here.

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FIN Newsletter October 2016

The October 2016 Fabry Newsletter is now available and can be downloaded here.

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Application open for Shire’s Humanitarian Programme

It is generally accepted that people affected by ultra-rare diseases across the globe face challenges getting reimbursed high cost market approved treatments. Like many LSD patient organisations across the World the MPS Society receives 100–200 emails each year from patients and their families pleading for help to access enzyme replacement therapy (ERT) for MPSI, MPSII, MPSIVA, MPSVI and Fabry … Continue reading “Application open for Shire’s Humanitarian Programme”

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FIN expert meeting – save the date!

Save the date! Due to the success of the FIN Fabry Expert meeting held this year in Tokyo, Japan, a date has now been secured for the 5th Fabry International Network Meeting 19th & 20th May 2017 Athens, Greece Further details to follow  

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