Ben Samelson-Jones, MD, PhD
@DrSamelsonJones
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Pediatric Hematologist | Gene Therapist | Biochemist | Husband | 👩👦👧👶 🐕 Dad | #WilliamsSyndrome Advocate | Views my own
Philadelphia, PA
Joined October 2019
RT @miesbach: Six-Year Gene Therapy Data for Haemophilia B - presented at ISTH 2025. 🔬 The remarkable long-term data from the phase 1/2a hi….
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RT @AmerMedicalAssn: Today is a sad and harmful day for patients and health care across the country, and its impact will reverberate for ye….
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RT @pjlynch: I found myself getting angry during a work meeting last week. Over coffee, a colleague told me about a skeptical physician wh….
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Amazing clinical development speed in #genetherapy.
🧬 BBM-H901: China's Regulatory Speed Breakthrough 🇨🇳 While most companies face 2-4 year delays, BBM-H901 got approval in just 18 months from pivotal study start! Great presentation at ISTH 2025 by Dr Feng Xue. 🤝 Belief BioMed 🇨🇳 + Takeda China 🇯🇵.China's Challenge: 235
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RT @ChildrensPhila: Congratulations to Dr. Leslie Raffini, Director of our Hemophilia Treatment Center, on delivering the Janna Journeycake….
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Medicaid is the backbone of children's healthcare in this country. If you do NOT want to live in a country where children are denied healthcare, click below to advocate . Add your voice to this campaign!
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Another ⬆️ impact #genetherapy study funded by Science Foundation of China. At #ISTH2025 and #ASGCT, there were numerous early phase studies from 🇨🇳. 🇺🇸is at risk of losing preeminence in biomedical research.
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RT @DrKiro_Kamel: RIETE registry data demonstrating that patients who retain their IVC filters have globally worse outcomes regardless of a….
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You need to meet 1️⃣ mom who passed Hep B to their child to never want this to happen again.
Hepatitis B can be passed from parent to baby at birth - and when that happens, the consequences can be deadly. It is unscientific & dangerous to ignore the success of U.S. vaccination programs or argue that the U.S. should not vaccinate babies for hepatitis B at birth.
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The reduced their maternal mortality by HALF! But still 20-100x rate in resource rich areas. 💔 thinking about how many lives could be saved with just some funding.
Absolutely incredible results from large >16k #women prospective study improving maternal morbidity & mortality through proactive interventions to prevent PPH #ISTH2025
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It is definitely dogma (pun intended) that gene addition is limited to long live cells in adults. However @paul_batty1 results at #ISTH2025 in infant #hemophilia A dogs is calling this into ?. We will present similar results in HB dogs at OC 69.3
Pipe #ISTH2025 Nice graphic on Gene Therapy ( Episomal,limited to long lived liver cells, adults only) v Gene Editing (Integrated in genome lasting effect, adults and children)
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Sustained and stable FIX levels have now been repeatedly been observed across multiple #genetherapy clinical trials for #hemophila B. Will extend the red line tomorrow at Clinical Gene Therapy session at #ISTH2025.
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What a great plenary lecture of Leslie Raffini at #ISTH2025 Pediatric Thrombosis: a lot has been done in 30 years, but still a lot to be done in the coming years!
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Brilliant visualization of extravascular FIX and very interesting (and surprising) differences in specific activity of extravascular FIX and FX in different tissues. 🤯. #ISTH2025
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RT @MarcCarrier1: 🩸 New insights from the RENOVE trial (extended duration anticoagulation) at #ISTH2025:. 📉 Reduced-dose DOAC is non-inferi….
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Potential emerging signal difference in ADAs in next-generation FVIII(a)-mimetics. 0/61 for Mim8 but 2/30 for NXT007. Surveillance may not be the same. #ISTH2025 #hemophilia
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#Hemophilia A patient treated with Hympavzi developed spontaneous DVT 3 years into treatment. Factor V Leiden heterozygous and sedentary lifestyle identified as risk factor. #ISTH2025
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