Blog
RSS FeedPosted by Michael Wonder on 20 Nov 2022
PHARMAC calls for applications for medicines for rare disorders
21 November 2022 - PHARMAC is calling for applications for medicines for rare disorders. ...
Posted by Michael Wonder on 06 Nov 2022
Asfotase alfa for the treatment of patients with paediatric-onset hypophosphatasia
4 November 2022 - The Department of Health and Social Care has asked NICE to review the guidance on the ...
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 ...
Posted by Michael Wonder on 31 Aug 2022
FDA approves first treatment for acid sphingomyelinase deficiency, a rare genetic disease
31 August 2022 - Today, the US FDA approved Xenpozyme (Olipudase alfa) for intravenous infusion in paediatric and adult patients with ...
Posted by Michael Wonder on 26 Aug 2022
Patient registries: EMA officials highlight opportunities in orphan drug development
25 August 2022 - Officials from the EMA tout the benefits of patient registries to support regulatory decision making for ...
Posted by Michael Wonder on 26 Jul 2022
‘No magic bullet’: For drug makers and the FDA, clinical trials on ultra rare diseases pose thorny challenges
26 July 2022 - Walker Burger is beside himself with worry. ...
Posted by Michael Wonder on 23 Jul 2022
Alnylam receives positive CHMP opinion for vutrisiran for the treatment of hereditary transthyretin-mediated amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy
22 July 2022 - Positive opinion based on HELIOS-A Phase 3 study. ...
Posted by Michael Wonder on 18 Jul 2022
Velmanase alfa for the treatment of alfa mannosidosis
15 July 2022 - The Department of Health and Social Care has asked NICE to produce guidance on using velmanase alfa ...
Posted by Michael Wonder on 07 Jul 2022
US FDA awards rare paediatric disease designation to paxalisib for AT/RT, a rare form of childhood brain cancer
6 July 2022 - Kazia Therapeutics is pleased to announce that the U.S. FDA has awarded rare paediatric disease designation ...