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On 10 March 2005, orphan designation (EU/3/05/264) was granted by the European Commission to TROPHOS SA, France for cholest-4-en-3-one, oxime for the treatment of 5 q spinal muscular atrophy.

The sponsorship was transferred to Roche Registration Limited, United Kingdom, in November 2015.

The sponsorship was transferred to Roche Registration GmbH, Germany in October 2018.

Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in April 2019 on request of the Sponsor.

Key facts

Active substance
cholest-4-en-3-one, oxime
Disease / condition
Treatment of 5q spinal muscular atrophy
Date of first decision
EU designation number

Sponsor's contact details

Roche Registration GmbH
Emil-Barell-Strasse 1
79639 Grenzach-Wyhlen
Tel. +49 7624 142892

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe;

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