Addenbrooke's Hospital
Biomedical Research
The Rosie Hospital
Lysosomal Disorders
Addenbrooke's Lysosomal Disorders Unit
Newsflash - Cerezyme/Fabrazyme shortage update
Update: 19 February 2010
Genzyme announce temporary closure of Boston manufacturing plant
> Read Genzyme press release (26 June 2009).
The information below has been updated to reflect the current situation.
- Fabrazyme: reduction in supplies anticipated to continue until Jun 2010. Please continue to follow instructions given to you by your centre
> Letter from hte manufacturer about Fabrazyme
- Cerezyme: supply has resumed but temporary restrictions apply. All patients will receive ½ dose for the next 2 months
> Letter from hte manufacturer about Cerezyme
- Addenbrooke's patients will all be contacted to advise them of their individual treatment plan
- It is likely that the shortage will continue and further rationing may be necessary
- UK Clinicians will continue to monitor the situation in conjunction with the Gaucher Association
- No safety concerns about any stock already supplied
- Call us on 01223 274 634 if you are worried
Genzyme Products, marketed as Cerezyme, Fabrayzme, Myozyme, Aldurazyme, and Thyrogen
Update: 19 February 2010
Cerezyme, Fabrazyme, Myozyme & Aldurazyme should now to be infused through a 0.2 micron filter.
For more information please read the information on the U.S. Food and Drug Administration website.
> Genzyme Products, Marketed as Cerezyme, Fabrayzme, Myozyme, Aldurazyme, and Thyrogen
Adult Services
Lysosomal Disorders Unit
Box 135
Tel: 01223 274 634
Fax: 01223 256 172
lysosomaldisordersunit@addenbrookes.nhs.uk
Children’s Services
Children’s Metabolic Unit
Box 181
Tel: 01223 216 878
Fax 01223 586 794
> Children’s Metabolic Unit contacts
Please note: The Children's Metabolic Unit web pages are currently being developed
Lysosomal storage diseases are a group of rare inherited metabolic disorders that are characterized by an abnormal build-up of various toxic materials in the body's cells as a result of enzyme deficiencies. There are nearly 50 of these altogether and new lysosomal storage disorders continue to be identified.
They can be classified by the nature of the primary stored material involved, and can be broadly broken into the following groups:
- Glycosphingolipidoses - Including Gaucher, Fabry, Sandhoff, Tay Sachs, Metachromatic Leukodystrophy & Niemann Pick A & B disease
- Mucopolysaccharidoses - MPS I (Hurler), MPS II (Hunter), MPS III (San Filippo), MPS IV (Morquio), MPS VI (Maroteaux Lamy) MPS VII (Sly)
- Glycoproteinoses - eg Schindler disease
- Other - eg Pompe, Danon, Niemann-Pick C, I Cell, Battens disease
Different diseases may affect various parts of the body, including the skeleton, brain, skin, heart, liver, kidney and central nervous system. Some of the lysosomal disorders now have licensed treatments available, and others are currently the subject of clinical and academic research. Symptom support and management is offered for those disorders currently without an approved treatment.
Addenbrooke's Hospital provides services for investigation and management of these diseases in both children (in the Children’s Metabolic Unit) and adults (in the Lysosomal Disorders unit).
Contact the Lysosomal Disorders Unit:
Box 135
Cambridge University Hospitals NHS Foundation Trust
Hills Road,
Cambridge,
CB2 0QQ
Tel: 01223 274 634
Fax: 01223 256 172
lysosomaldisordersunit@
addenbrookes.nhs.uk
From Addenbrooke's news:
> Leading artist donates work to Addenbrooke’s
The family of celebrated artist Hugh Mackinnon have generously donated a landmark work, which is now on public display in the Lysosomal Disorders Unit.
