29 February 2024 - On Rare Disease Day — a day when the international community raises awareness about rare conditions ...
29 February 2024 - PHARMAC understands that people living with rare disorders face many challenges, including access to health care ...
18 January 2024 - Since July 2023, there has been one in Germany for patients who suffer from acute myeloid leukaemia ...
10 January 2023 - Sebelipase alfa is recommended as an option for the treatment of patients with Wolman disease only if ...
13 December 2023 - NICE has published final evidence-based recommendations on the use of velmanase alfa (Lamzede) for treatment of ...
13 November 2023 - PHARMAC is calling for applications for medicines to treat rare disorders. ...
3 November 2023 - The Department of Health and Social Care has asked NICE to produce guidance on the use ...
26 October 2023 - The Department of Health and Social Care has asked NICE to produce guidance on the use ...
15 August 2023 - NICE has published evidence-based recommendations on the use of cipaglucosidase alfa (Pombiliti) with miglustat (Opfolda) for ...
10 August 2023 - PHARMAC has published the record of the March Rare Disorders Advisory Committee meeting, sharing details on ...
3 August 2023 - The Department of Health and Social Care has asked NICE to produce guidance on the use ...
10 May 2023 - A team led by IQWiG authors, together with Rita Banzi from the Italian Mario Negri Institute, analysed ...
11 April 2023 - The Australian Government has added the two medicines, Kanuma (sebelipase alfa) and Cerdelga (eliglustat) to the Life ...
22 March 2023 - Today, the Government announced a total investment of up to $1.5 billion over three years in ...
22 March 2023 - Today, the Honourable Jean-Yves Duclos, Minister of Health, announced measures in support of the first-ever National Strategy ...