Blog
RSS FeedPosted by Michael Wonder on 23 Mar 2019
FDA takes new steps to advance natural history studies for accelerating novel treatments for rare diseases
22 March 2019 - The U.S. FDA today issued the draft guidance, Rare Diseases: Natural History Studies for Drug Development. ...
Posted by Michael Wonder on 21 Mar 2019
Is Orkambi deal close? Vertex meets again today with NHS England
21 March 2019 - Campaigners hopes rise after three year wait. ...
Posted by Michael Wonder on 12 Mar 2019
Vertex receives approval for Symdeko (tezacaftor/ivacaftor and ivacaftor) in Australia, to treat the underlying cause of cystic fibrosis in people aged 12 and older with certain CFTR gene mutations
12 March 2019 - A new treatment option for patients with two copies of the F508del mutation, the most common mutation ...
Posted by Michael Wonder on 12 Mar 2019
Optimism on Orkambi in England as talks to resume
12 March 2019 - Scotland's deal puts pressure on NHS England and NICE. ...
Posted by Michael Wonder on 11 Mar 2019
Cystic fibrosis breakthrough: vital drug could be made available as early as next month
8 March 2019 - A wonder drug that could dramatically improve the lives of thousands of cystic fibrosis patients may be ...
Posted by Michael Wonder on 08 Mar 2019
Campaigners call for NHS access to Orkambi as MP inquiry gets underway
8 March 2019 - Patients and campaigners took to the streets in London yesterday demanding NHS access to Vertex’ cystic ...
Posted by Michael Wonder on 08 Mar 2019
High price of cystic fibrosis drug not political, firm's boss tells MPs
8 March 2019 - CEO of company that makes Orkambi denies being ‘political minion’ for Donald Trump. ...
Posted by Michael Wonder on 06 Mar 2019
In UK and Ireland, SMA patients demand reimbursement for Spinraza
6 March 2019 - A drug appraisal committee of Britain’s NICE met in Manchester, England, for the third and final ...
Posted by Michael Wonder on 06 Mar 2019
FDA grants rare paediatric disease designation to odiparcil for the treatment of MPS VI
5 March 2019 - Inventiva eligible to receive priority review voucher upon approval of odiparcil for the treatment of MPS VI. ...
Posted by Michael Wonder on 05 Mar 2019
How to fix a problem like Orkambi? Solutions suggested ahead of showdown
5 March 2019 - Approaches used in France and Germany could help break deadlock. ...
Posted by Michael Wonder on 05 Mar 2019
FDA is working to bridge gaps and meet needs for rare disease product development
28 February 2019 - There are approximately 7,000 rare diseases affecting an estimated 30 million people in the United States. ...
Posted by Michael Wonder on 04 Mar 2019
Vertex chief Jeffrey Leiden to weigh in on Orkambi inquiry
4 March 2019 - Vertex’s long-running spat with NHS England has amounted to a legal inquiry scheduled to begin this Thursday, ...
Posted by Michael Wonder on 04 Mar 2019
Akcea and Ionis receive positive EU CHMP opinion for Waylivra (volanesorsen)
1 March 2019 - First and only therapy approved for FCS, a serious and rare disease with no approved treatment options. ...
Posted by Michael Wonder on 04 Mar 2019
BioMarin receives positive CHMP opinion in Europe for Palynziq (pegvaliase injection) for treatment of patients with phenylketonuria aged 16 and older
1 March 2019 - Decision on marketing authorisation application expected Q2 '19. ...
Posted by Michael Wonder on 26 Feb 2019
They can’t get their medicines. So they’re moving to Scotland.
26 February 2019 - Families in England are preparing to leave the country in a desperate bid to obtain a cystic ...