Joined September 2013
- Tweets 161
- Following 152
- Followers 538
- Likes 99
29 Photos and videos
Mark Skinner retweeted
6 Sep 2025
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.
4
9
32
1,100
Mark Skinner retweeted
12 Aug 2025
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
3
1
12
1,200
18 Jul 2025
Why is the uptake of gene therapy in hemophilia less than expected? rpthjournal.org/article/S247…
Please review our current article detailing the complex decision making process, one that goes well beyond "patient interest". The desire to be cured remains strong.
2
8
111
10 Jul 2025
We’re deeply saddened by the passing of Drs. Pattraporn Isarangkura & Carol Kasper—true giants in our community. Appropriately jointly named as the 1st recipients of the @wfhemophilia Volunteer Award (2004). We honor their legacy with deep gratitude—their impact will live on.
1
1
6
134
Mark Skinner retweeted
8 Mar 2025
✨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
6
14
446
Mark Skinner retweeted
6 Dec 2024
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
1
7
11
480
Mark Skinner retweeted
6 Dec 2024
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
1
7
10
510
Mark Skinner retweeted
6 Dec 2024
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
1
10
25
1,481
Mark Skinner retweeted
8 Dec 2024
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
1
16
29
1,998
Mark Skinner retweeted
9 Dec 2024
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
9
15
521
9 Dec 2024
Contrasting Approaches in the Implementation of GRADE Methodology in Guidelines for Haemophilia and VWD. onlinelibrary.wiley.com/doi/… This article developed by a global team of 48 experts and international organizations rigorously examines a number of GRADE methodology adaptations.
1
2
7
735
9 Dec 2024
The puristic approach of the ISTH Guideline development, without consideration of accepted adaptations to GRADE implementation, led to recommendations that have been widely deemed invalid and obsolete by expert healthcare professionals and those living with hemophilia.
1
2
64
9 Dec 2024
The methodology should have allowed data from registries and real-world studies, integrated multiple patient-related outcomes and expert opinion. The strict application led ISTH to limited, conservative, and unambitious guidelines failing to consider newer treatments.
3
48
Mark Skinner retweeted
26 Jun 2023
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
1
1
4
568
Mark Skinner retweeted
26 Jun 2023
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
1
16
56
9,276
13 May 2023
The Aging with a bleeding disorder panel at #WFHCCS presented many new challenges. A specialist multidisciplinary team is needed to ensure all individuals receive appropriate individualized care. @WillMcKeown Felipe Contreras, Sue Cutter, Piet de Klein @lochibabes @NiamhLarkinANP
4
7
39
1,722
12 May 2023
Pleased to report Initial Results of the Impact of
Valoctocogene Roxaparvovec on Pain
Occurrence and Interference:
Insights from PROBE at #wfhCCS. Chronic pain is a core patient-important outcome.
3
13
1,677
Mark Skinner retweeted
10 May 2023
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
4
7
646
Mark Skinner retweeted
10 May 2023
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
1
3
8
422
Mark Skinner retweeted
10 May 2023
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
3
8
456