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Cyclodextrine (Hydroxypropyl betadex)

Extended indication Ziekte van Niemann-Pick, type C
Therapeutic value No judgement
Registration phase Clinical trials

Product

Active substance Cyclodextrine (Hydroxypropyl betadex)
Domain Metabolism and Endocrinology
Main indication Metabolic diseases
Extended indication Ziekte van Niemann-Pick, type C
Manufacturer Vtesse
Route of administration Intrathecal
Therapeutical formulation Injection
Budgetting framework Intermural (MSZ)

Registration

Registration route Centralised (EMA)
Particularity New medicine
Orphan drug Yes
Registration phase Clinical trials

Therapeutic value

Therapeutic value No judgement

Expected patient volume per year

References Erfelijkheid.nl; Vanier et al. Orphanet J Rare Dis. 2010 Jun 3;5:16
Additional remarks De incidentie van Niemann-Pick type C is 1:250.000, wat neerkomt op 68 patiënten in Nederland. De incidentie bij pasgeborenen is 1:120.000, dit is dus 1-2 nieuwe patiënten per jaar

Expected cost per patient per year

There is currently nothing known about the expected cost.

Potential total cost per year

There is currently nothing known about the possible total cost.

Off label use

There is currently nothing known about off label use.

Indication extension

There is currently nothing known about indication extensions.

Other information

There is currently no futher information available.