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FOI 0914 2020/21 Prescribing in Inherited Metabolic Disorders

Freedom of Information Request: 0914 2020/21

We have a freedom of information request related to the incidence and treatment of some inherited metabolic disorders. Could you please provide answers to the following four questions?

Q1. Within your trust, how many patients currently have a diagnosis for

Fabry Disease – 151

Gaucher Disease – 14

MPS II (Hunter Syndrome) – 4

Q2. Of the patients above, how many have been newly diagnosed within the past 3 months for

Fabry Disease – 0

Gaucher Disease – 0

MPS II (Hunter Syndrome) – 0

Q3. How many patients (including any patients whose treatment is on hold due to Covid-shielding) are being treated at this point in time with:

Replagal (agalsidase alpha) – 24

Fabrazyme (agalsidase beta) – 15

Galafold (migalastat)   – 31

VPRIV (velaglucerase alfa) – 1        

Cerezyme (imiglucerase)   – 3

Cerdelga (eliglustat)   – 9

Zavesca (miglustat) – 1

Any other treatment for Fabry disease [please state which treatments] Not Applicable

Any other treatment for Gaucher disease [please state which treatments] Not Applicable  

Q4. Of the patients being treated for Fabry disease can you please provide the numbers of patients being treated through homecare?


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