Blog
RSS FeedPosted by Michael Wonder on 05 Mar 2023
Our progress in the funding of medicines for rare disorders
3 March 2023 - PHARMAC will hear from people affected by rare disorders in its upcoming Rare Disorders Advisory Committee meeting ...
Posted by Michael Wonder on 02 Mar 2023
Asfotase alfa for the treatment of certain patients with paediatric-onset hypophosphatasia
1 March 2023 - This evaluation reviews the evidence for asfotase alfa for the treatment of patients with paediatric‑onset hypophosphatasia (HST6), ...
Posted by Michael Wonder on 28 Feb 2023
My son’s time is running out due to a rare disease. The FDA needs to add more clinical trial flexibility.
28 February 2023 - My toddler, Wheeler, will probably not live to adulthood. Juvenile Batten disease — he has the ...
Posted by Michael Wonder on 24 Feb 2023
Chiesi Global Rare Diseases and Protalix BioTherapeutics receive positive CHMP opinion for pegunigalsidase alfa for treatment of Fabry disease
24 February 2023 - European Commission decision anticipated in beginning of May 2023. ...
Posted by Michael Wonder on 23 Feb 2023
FDA continues important work to advance medical products for patients with rare diseases
23 February 2023 - Patients with rare diseases are experts in their health condition. They provide a unique perspective, and ...
Posted by Michael Wonder on 17 Feb 2023
Chiesi Global Rare Diseases announces FDA approval of Lamzede (velmanase alfa-tycv) for alfa mannosidosis
16 February 2023 - First and only enzyme replacement therapy for the treatment of non-central nervous system manifestations of alfa mannosidosis ...
Posted by Michael Wonder on 28 Jan 2023
Ipsen receives CHMP negative opinion for palovarotene as a treatment for fibrodysplasia ossificans progressiva in EU
27 January 2023 - Ipsen to request re-examination of CHMP opinion on palovarotene as a potential treatment for fibrodysplasia ossificans progressiva ...
Posted by Michael Wonder on 23 Jan 2023
Despite court ruling, FDA will continue with its approach to approving orphan drug exclusivity
23 January 2023 - In an unexpected move, the FDA will continue to apply exclusive marketing rights for so called ...
Posted by Michael Wonder on 06 Nov 2022
Kiwis with rare disorders want faster progress on 'life or death' health strategy
6 November 2022 - For Samantha Lenik, being diagnosed with Pompe disease after seven years of symptoms was a “double-edged ...
Posted by Michael Wonder on 04 Oct 2022
Rare disease endpoint advancement pilot program
3 October 2022 - FDA is establishing a Rare Disease Endpoint Advancement (RDEA) Pilot Program to support novel efficacy endpoint development ...
Posted by Michael Wonder on 18 Sep 2022
Agios receives positive CHMP opinion for Pyrukynd (mitapivat) for the treatment of pyruvate kinase deficiency in adult patients
16 September 2022 - Agios expects a decision on the marketing authorisation application by the European Commission within 67 days of ...
Posted by Michael Wonder on 12 Sep 2022
Chiesi Global Rare Diseases announces FDA acceptance of BLA filing for velmanase alfa for the proposed treatment of alfa mannosidosis
12 September 2022 - FDA grants priority review designation with PDUFA date in the first half of 2023. ...
Posted by Michael Wonder on 09 Sep 2022
Rare cancer sufferers fight to get drugs on PBS
9 September 2022 - People with rare cancers are being forced to sell their homes, raid their superannuation and fundraise ...
Posted by Michael Wonder on 07 Sep 2022
The FDA needs to be more flexible in assessing treatments for rare diseases, like the one that seemed to help my son
7 September 2022 - Every time I read about clinical trials testing possible treatments for rare diseases, I think of ...
Posted by Michael Wonder on 04 Sep 2022
Australia among world's best in treating debilitating and often fatal muscle disorder
4 September - Australia has become one of the first countries in the world to fund a new, next-generation treatment ...