Discover and read the best of Twitter Threads about #haemophilia
Most recents (3)
Although the list or gross price of Roctavian, the new hemophilia A gene therapy, has not been decided/made public, we have information on the net price that the manufacturer expects from each dose sold: 1.5million euro. #hemophilia #haemophilia 1/3
The price will be based on Outcomes Based Agreement for 5-8 years. Payment assumes the patient is not back on prophylaxis. Example: 5 year agreement. Payment upfront. If patient goes back on prophylaxis after 4 years, there will be a 20% refund. 2/3
The main target for the new hemophilia A gene therapy is Germany. The 1.5m euro price is based on the cost of the current hemophilia therapy in Germany. 3/3 #hemophilia
Thread
Through 2021 young people living with #RareDiseases share their experience of transitioning from paediatric to adult care in this new report created by Costello Medical in collaboration with @camraredisease & @RareBeacon
Please download & share lnkd.in/e4zc5WZ6
Through 2021 young people living with #RareDiseases share their experience of transitioning from paediatric to adult care in this new report created by Costello Medical in collaboration with @camraredisease & @RareBeacon
Please download & share lnkd.in/e4zc5WZ6
Transition has been described as “the purposeful and planned movement of adolescents and young adults with chronic physical and medical conditions from child-centred to adult orientated healthcare systems”.
But were their experiences planned & purposeful?
#Transition
But were their experiences planned & purposeful?
#Transition
In the UK Rare Diseases Framework published Jan ‘21, it’s recognised that we must address “the challenges in ensuring continuity of care across paediatric and adult services” to reduce the burden of care on patients and their families.
@DHSCgovuk @GeneticAll_UK #CONCORDstudy
@DHSCgovuk @GeneticAll_UK #CONCORDstudy
I joined the Biomarin conference this evening. Not much more info than the press release but here are a few snippets that I learned. 1/7
Biomarin phase 3 steroid use. Number of patients who needed steroids not mentioned. No mention of steroid adverse events. They said they just completed recruitment of a study that uses prophylactic steroids. 2/7
I believe that in #hemophilia gene therapy studies the adverse events due to steroids should be mentioned. These are high dose steroids for several weeks and adverse events are common. 3/7
