Mark Skinner retweeted
The global #BleedingDisorders community is celebrating a milestone achievement! The #WHO has approved the WFH proposals to update the Essential Medicines List and the Essential Medicines List for Children. Stay tuned for more news on the work carried out by the WFH.
Mark Skinner retweeted
Thank you for having me @VJHemOnc
Several colleagues and I also published a reflective article on this:
tinyurl.com/Why-is-the-uptak…
I summarized some key points in the video.
SPOILER ALERT
It is not due to lack of interest in gene therapy.
#hemophilia
Why has the uptake of gene therapy in #hemophilia been less than expected? Is there limited interest in these therapies? 🤔🩸🧬
We caught up with @RKaczmarekDr of @IUMedSchool to get the answers to these questions:
👉 ow.ly/LFKL50WAwFA 👈
#GeneTherapy #Hematology #ISTH2025
Mark Skinner retweeted
✨This #IWD2025, let’s unite to drive change! The PROBE Study is now adapted for women with bleeding disorders, helping to gather data, improve diagnosis, and advocate for better treatment. 📢 Participate: bit.ly/41urMt9 #WeBleedToo
Mark Skinner retweeted
Process flawed with methodologist and non haemophilia treaters in lead roles. Patients initially involved all withdrew from the process prior to publication. An additional paper to follow critiquing the methodology. @wfhemophilia @EAHADnews @EHC_Haemophilia @HaemophiliaIRL 3/3
Mark Skinner retweeted
Conclusions: Methodological approach used resulted in categorisation of evidence as weak. Failure to recognise the advantages of modern therapies. May misguide treaters, payers and Governments as they overlook important evidence and incorporate obsolete treatment principles 2/3
Mark Skinner retweeted
A critique of the ISTH Haemophilia Guidelines was published in #Haemophilia Journal today with 49 eminent authors representing a global mixture of expertise, all united in their serious concerns about these guidelines which were already outdated on publication 1/3
Mark Skinner retweeted
The WFH supports the findings of 49 global experts that examined the ISTH Guidelines & published a critical appraisal in @haemophilia_jnl. The appraisal highlights issues in the ISTH publication that could impact health outcomes for people with hemophilia: bit.ly/4g1zP70
Mark Skinner retweeted
The WFH is aligned with the findings of 48 global experts within the #BleedingDisorder community, that have come together to publish an article on contrasting approaches in applying the GRADE methodology and how it can impact medical guidelines: bit.ly/3CWeQ6L
Mark Skinner retweeted
In the paper, we review the data leading to the regulatory authorization in Europe of valoctocogene roxaparvovec, an adeno-associated virus (AAV)-5 gene therapy to treat haemophilia A and etranacogene dezaparvovec in the United States and Europe to treat haemophilia B. 2/3
Mark Skinner retweeted
Paper just published online. The decision to take a gene therapy product is one in which an informed, holistic and shared decision-making approach must be employed. Bias on the part of healthcare professionals and people with haemophilia must be addressed and minimized. 1/3
Mark Skinner retweeted
Recommendations for future of ageing PWH: recognise variation, impact on all genders, changed time horizons lead to new social rules, need enhanced mental health support #wfhCCS @MSkinnerDC
Mark Skinner retweeted
Australian PROBE data shows impact of haemophilia on employment. In PWH 45-64 years, 50% in fulltime employment v over 80% in control population. For those aged >65, <10% of PWH employed full time v almost 30% for controls #wfhCCS @MSkinnerDC
Mark Skinner retweeted
Australian PROBE data shows continued impairment in men with haemophilia in physical functioning and levels of pain compared to men with no bleeding disorder. @MSkinnerDC @wfhemophilia #wfhCCS
Mark Skinner retweeted
@MSkinnerDC outlines key members of the comp care team and points out increased need for expertise of Cardiologists, Gerontologists and others when Ageing with Haemophilia. He mentioned Dentists who should be on the core team in any case as they are vital at all ages #wfhCCS
Mark Skinner retweeted
On this #WorldHaemophiliaDay we spoke to Professor Johnny Mahlangu about treatment and unmet needs of those living with the disease. Patient advocate @MSkinnerDC also talks about his experience of living with #haemophilia.
Watch here: ow.ly/Ik8U50NKnQf
Mark Skinner retweeted
Don't miss the @TheLancetHaem article on the WFH Humanitarian Aid Program and how it’s facilitating access to care for people with #BleedingDisorders in developing countries. Read below 👇
ICYMI "The WFH HAP is an exemplar of partnership between for-profit and not-for-profit organisations advancing health-care equity in... LMICs"
🌐 Policy review on the @wfhemophilia's expanded Humanitarian Aid Program #hemophilia #bleedingdisorders thelancet.com/journals/lanha…
Mark Skinner retweeted
A new paper features a helpful Q&A resource on shared decision making and gene therapy. Learn more: https://nitter.gq/t.co/VxgDtUF4h6
Mark Skinner retweeted
Kenya has shown me that we can improve the health of people with hemophilia in Africa. These children told me that they were proud to have hemophilia. They are WORLD FEDERATION OF HEMOPHILIA.@wfhemophilia @WFHUSA @EHC_Haemophilia @coalicionameric @hemnet
Mark Skinner retweeted
On 29th July 2022,
@wfhemophilia President Cesar Garrido, @KenyaHaemophil4 President Dr Kibet Shikuku and Ministry of Health officials led by CAS Rashid Aman held discussions on the current state of affairs for patients with Hemophilia and other bleeding disorders in Kenya
