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 ...
10 March 2023 - Company expects Daybue to be available by the end of April 2023. ...
7 March 2023 - Genethon today announced that the EMA has granted PRIME (Priority Medicines) status to the gene therapy, ...
3 March 2023 - PHARMAC will hear from people affected by rare disorders in its upcoming Rare Disorders Advisory Committee meeting ...
1 March 2023 - This evaluation reviews the evidence for asfotase alfa for the treatment of patients with paediatric‑onset hypophosphatasia (HST6), ...
28 February 2023 - My toddler, Wheeler, will probably not live to adulthood. Juvenile Batten disease — he has the ...
27 February 2023 - PHARMAC is creating visibility of what medicines are being considered that could impact the rare disorders ...
24 February 2023 - European Commission decision anticipated in beginning of May 2023. ...
23 February 2023 - Patients with rare diseases are experts in their health condition. They provide a unique perspective, and ...
16 February 2023 - First and only enzyme replacement therapy for the treatment of non-central nervous system manifestations of alfa mannosidosis ...
27 January 2023 - Ipsen to request re-examination of CHMP opinion on palovarotene as a potential treatment for fibrodysplasia ossificans progressiva ...
23 January 2023 - In an unexpected move, the FDA will continue to apply exclusive marketing rights for so called ...
6 November 2022 - For Samantha Lenik, being diagnosed with Pompe disease after seven years of symptoms was a “double-edged ...
3 October 2022 - FDA is establishing a Rare Disease Endpoint Advancement (RDEA) Pilot Program to support novel efficacy endpoint development ...