Signal or noise? > Sickle Cell Disease and β-Thalassemia Gene Therapy Market
Set - openPR.com >> Comment below! #strategy #competitiveintelligence #marketing #healthcare #pharmaceutical #competitivemarketing #biotech #pharma
Set - openPR.com >> Comment below! #strategy #competitiveintelligence #marketing #healthcare #pharmaceutical #competitivemarketing #biotech #pharma
Sickle Cell Disease and β-Thalassemia Gene Therapy Market
Set - openPR.com
Sickle Cell Disease and β-Thalassemia Gene Therapy Market Set openPR.com
dlvr.it
February 19, 2026 at 10:29 AM
Signal or noise? > Sickle Cell Disease and β-Thalassemia Gene Therapy Market
Set - openPR.com >> Comment below! #strategy #competitiveintelligence #marketing #healthcare #pharmaceutical #competitivemarketing #biotech #pharma
Set - openPR.com >> Comment below! #strategy #competitiveintelligence #marketing #healthcare #pharmaceutical #competitivemarketing #biotech #pharma
Turns out that there's a whole genre of AI flimflam, claiming to have trained a ML tool on the same data-set so it could distinguish healthy carriers of ß-thalassemia from non-carriers with 95%+ accuracy. None mention consent or explain how they acquired the data.
scholar.google.com/scholar?hl=e...
scholar.google.com/scholar?hl=e...
February 18, 2026 at 12:48 AM
Turns out that there's a whole genre of AI flimflam, claiming to have trained a ML tool on the same data-set so it could distinguish healthy carriers of ß-thalassemia from non-carriers with 95%+ accuracy. None mention consent or explain how they acquired the data.
scholar.google.com/scholar?hl=e...
scholar.google.com/scholar?hl=e...
It seems that Punjab Province is populated with zombies & vampires. Also, the authors somehow gained access to intimate personal health records from a ß-thalassemia tracking program - no ethics approval or consent. Or (boring explanation) author Anas Bilal made it all up.
pubpeer.com/search?q=%22...
pubpeer.com/search?q=%22...
PubPeer - Search publications and join the conversation.
PubPeer enables scientists to search for their publications or their peers publications and provide feedback and/or start a conversation anonymously.
pubpeer.com
February 18, 2026 at 12:35 AM
It seems that Punjab Province is populated with zombies & vampires. Also, the authors somehow gained access to intimate personal health records from a ß-thalassemia tracking program - no ethics approval or consent. Or (boring explanation) author Anas Bilal made it all up.
pubpeer.com/search?q=%22...
pubpeer.com/search?q=%22...
Their Mutated Genes Were Supposed to be Harmless: "Carriers" of certain genetic diseases, who have just one affected gene, can have symptoms too
Anecdotally, I've seen this during pregnancy, when "asymptomatic" thalassemia & sickle cell carriers may develop worrisome symptoms for the first time
Anecdotally, I've seen this during pregnancy, when "asymptomatic" thalassemia & sickle cell carriers may develop worrisome symptoms for the first time
Rare disease piece in @theatlantic.com, how carriers of recessive conditions can have medical problems, making them more than 'carriers'. Gift link below.
www.theatlantic.com/health/2026/...
www.theatlantic.com/health/2026/...
Their Mutated Genes Were Supposed to Be Harmless
“Carriers” of certain genetic diseases, who have just one affected gene, can have symptoms too.
www.theatlantic.com
February 16, 2026 at 5:36 PM
Their Mutated Genes Were Supposed to be Harmless: "Carriers" of certain genetic diseases, who have just one affected gene, can have symptoms too
Anecdotally, I've seen this during pregnancy, when "asymptomatic" thalassemia & sickle cell carriers may develop worrisome symptoms for the first time
Anecdotally, I've seen this during pregnancy, when "asymptomatic" thalassemia & sickle cell carriers may develop worrisome symptoms for the first time
Romosozumab for the treatment of thalassemia-associated osteoporosis in two female patients
Maria P Yavropoulou, Maria-Evangelia Koloutsou, Polyxeni Delaporta, Polyzois Makras, Athanasios D Anastasilakis, Eva Kassi, Antonis Kattamis, Paraskevi Xekouki
academic.oup.com/jbmrplus/adv...
Maria P Yavropoulou, Maria-Evangelia Koloutsou, Polyxeni Delaporta, Polyzois Makras, Athanasios D Anastasilakis, Eva Kassi, Antonis Kattamis, Paraskevi Xekouki
academic.oup.com/jbmrplus/adv...
February 16, 2026 at 3:41 PM
Romosozumab for the treatment of thalassemia-associated osteoporosis in two female patients
Maria P Yavropoulou, Maria-Evangelia Koloutsou, Polyxeni Delaporta, Polyzois Makras, Athanasios D Anastasilakis, Eva Kassi, Antonis Kattamis, Paraskevi Xekouki
academic.oup.com/jbmrplus/adv...
Maria P Yavropoulou, Maria-Evangelia Koloutsou, Polyxeni Delaporta, Polyzois Makras, Athanasios D Anastasilakis, Eva Kassi, Antonis Kattamis, Paraskevi Xekouki
academic.oup.com/jbmrplus/adv...
Thalassemia, CF, FMF, fragile X, and so on, found across the entire region. You poor thang
February 13, 2026 at 10:24 PM
Thalassemia, CF, FMF, fragile X, and so on, found across the entire region. You poor thang
#WIMMReads
🧪 #MRCWIMM Professor Doug Higgs authors a commentary in Blood on how chromatin remodeling complexes regulate globin genes and what this could mean for reactivating fetal hemoglobin in beta thalassemia and sickle cell disease.
#Haematology #GeneRegulation #WIMM
🧪 #MRCWIMM Professor Doug Higgs authors a commentary in Blood on how chromatin remodeling complexes regulate globin genes and what this could mean for reactivating fetal hemoglobin in beta thalassemia and sickle cell disease.
#Haematology #GeneRegulation #WIMM
Switching and Sniffing around the β-globin cluster
In this issue of Blood, Bagchi et al1 show how the β-like globin genes may be regulated by “Switch-Sniff” (switch/sucrose nonfermentable) chromatin remodel
ashpublications.org
February 12, 2026 at 10:24 AM
#WIMMReads
🧪 #MRCWIMM Professor Doug Higgs authors a commentary in Blood on how chromatin remodeling complexes regulate globin genes and what this could mean for reactivating fetal hemoglobin in beta thalassemia and sickle cell disease.
#Haematology #GeneRegulation #WIMM
🧪 #MRCWIMM Professor Doug Higgs authors a commentary in Blood on how chromatin remodeling complexes regulate globin genes and what this could mean for reactivating fetal hemoglobin in beta thalassemia and sickle cell disease.
#Haematology #GeneRegulation #WIMM
US Alpha Thalassemia Market Healthcare Systems Data 2035 www.marketresearchfuture.com/reports/us-a...
US Alpha Thalassemia Market Healthcare Systems Data 2035
US Alpha Thalassemia Market share register 1020.19 USD Million in 2024, projected to grow 10.16 % CAGR to reach USD Million 2957.64 during the forecast period 2025 - 2035.
www.marketresearchfuture.com
February 10, 2026 at 12:55 PM
US Alpha Thalassemia Market Healthcare Systems Data 2035 www.marketresearchfuture.com/reports/us-a...
Alpha Thalassemia Market Size, Trends, Growth Report 2035 www.marketresearchfuture.com/reports/alph...
Alpha Thalassemia Market Size, Trends, Growth Report 2035
Alpha Thalassemia Market share is projected to reach USD 12.07 Billion By 2035, at a 10.16 % CAGR by driving industry size, top company analysis, segments research, trends and forecast report 2025 to ...
www.marketresearchfuture.com
February 10, 2026 at 12:44 PM
Alpha Thalassemia Market Size, Trends, Growth Report 2035 www.marketresearchfuture.com/reports/alph...
Romosozumab can be an effective and safe anabolic option for patients with thalassemia and severe osteoporosis. MP Yavropoulou et al. JBMR Plus 5 Feb 2026 shorturl.at/YX9J6 … case reports of two female patients
February 9, 2026 at 9:50 AM
Romosozumab can be an effective and safe anabolic option for patients with thalassemia and severe osteoporosis. MP Yavropoulou et al. JBMR Plus 5 Feb 2026 shorturl.at/YX9J6 … case reports of two female patients
Thalassemia free Pakistan remains national goal, says Dr Jamal Nasir
- Advertisement - RAWALPINDI, Feb 06 (APP):The Pa
https://en.tezkhabar.tv/thalassemia-free-pakistan-remains-national-goal-says-dr-jamal-nasir/
#TezkhabarNews #Tezkhabar #News #Live #PakistanNews #Pakistan #Headline #NewsHeadline
- Advertisement - RAWALPINDI, Feb 06 (APP):The Pa
https://en.tezkhabar.tv/thalassemia-free-pakistan-remains-national-goal-says-dr-jamal-nasir/
#TezkhabarNews #Tezkhabar #News #Live #PakistanNews #Pakistan #Headline #NewsHeadline
February 6, 2026 at 11:25 AM
Thalassemia free Pakistan remains national goal, says Dr Jamal Nasir
- Advertisement - RAWALPINDI, Feb 06 (APP):The Pa
https://en.tezkhabar.tv/thalassemia-free-pakistan-remains-national-goal-says-dr-jamal-nasir/
#TezkhabarNews #Tezkhabar #News #Live #PakistanNews #Pakistan #Headline #NewsHeadline
- Advertisement - RAWALPINDI, Feb 06 (APP):The Pa
https://en.tezkhabar.tv/thalassemia-free-pakistan-remains-national-goal-says-dr-jamal-nasir/
#TezkhabarNews #Tezkhabar #News #Live #PakistanNews #Pakistan #Headline #NewsHeadline
if you really want an iron rich dinner rare liver and spinach. i had to eat that at least times a week as a kid because i had thalassemia (a genetic, inherited disorder causing severe anemia) lentils are also very high in iron had to eat those multiple times a week as well
February 3, 2026 at 1:17 AM
if you really want an iron rich dinner rare liver and spinach. i had to eat that at least times a week as a kid because i had thalassemia (a genetic, inherited disorder causing severe anemia) lentils are also very high in iron had to eat those multiple times a week as well
New from the #Transfusion Evidence Alert:
Safety and pharmacodynamics of the ferroportin inhibitor vamifeport in patients with non-transfusion-dependent β-thalassemia: results from a randomized phase 2a study
by Kattamis et al.
www.transfusionevidencelibrary.com/articles/412...
#MedSky
Safety and pharmacodynamics of the ferroportin inhibitor vamifeport in patients with non-transfusion-dependent β-thalassemia: results from a randomized phase 2a study
by Kattamis et al.
www.transfusionevidencelibrary.com/articles/412...
#MedSky
February 2, 2026 at 1:49 PM
New from the #Transfusion Evidence Alert:
Safety and pharmacodynamics of the ferroportin inhibitor vamifeport in patients with non-transfusion-dependent β-thalassemia: results from a randomized phase 2a study
by Kattamis et al.
www.transfusionevidencelibrary.com/articles/412...
#MedSky
Safety and pharmacodynamics of the ferroportin inhibitor vamifeport in patients with non-transfusion-dependent β-thalassemia: results from a randomized phase 2a study
by Kattamis et al.
www.transfusionevidencelibrary.com/articles/412...
#MedSky
I hear you. Competitive culture walks a thin line with ableism. I grew up thinking my consistent poor performance in athletic competition was a character flaw, when it was undiagnosed thalassemia and ADHD. “It’s just a game” they’ll say, but it never is.
January 31, 2026 at 2:14 PM
I hear you. Competitive culture walks a thin line with ableism. I grew up thinking my consistent poor performance in athletic competition was a character flaw, when it was undiagnosed thalassemia and ADHD. “It’s just a game” they’ll say, but it never is.
"Many patient populations with hereditary conditions such as #thalassemia have lived w the disease for their entire life, so it’s not surprising that they report high levels of confidence in health-related knowledge" @devasharma.bsky.social of @vumedicine.bsky.social.
https://bit.ly/4t4hyN6
#ASH25
https://bit.ly/4t4hyN6
#ASH25
Significant Barriers to Health Literacy Exist for Patients With Thalassemia
Researchers determined there may be overestimation of disease understanding and barriers to health literacy among patients with thalassemia.
bit.ly
January 27, 2026 at 8:35 PM
"Many patient populations with hereditary conditions such as #thalassemia have lived w the disease for their entire life, so it’s not surprising that they report high levels of confidence in health-related knowledge" @devasharma.bsky.social of @vumedicine.bsky.social.
https://bit.ly/4t4hyN6
#ASH25
https://bit.ly/4t4hyN6
#ASH25
nope!! it’s called thalassemia and if you have the minor form of it you’re completely asymptomatic
January 27, 2026 at 5:54 PM
nope!! it’s called thalassemia and if you have the minor form of it you’re completely asymptomatic
We're excited to hear from 𝐃𝐫 𝐌𝐢𝐭𝐜𝐡𝐞𝐥𝐥 𝐖𝐞𝐢𝐬𝐬, whose lab at St Jude Children's Research Hospital investigates red blood cell biology to develop new treatments for sickle cell disease and β-thalassemia, conditions affecting millions worldwide: https://www.lornegenome.org/mitch-weiss
January 27, 2026 at 12:12 AM
We're excited to hear from 𝐃𝐫 𝐌𝐢𝐭𝐜𝐡𝐞𝐥𝐥 𝐖𝐞𝐢𝐬𝐬, whose lab at St Jude Children's Research Hospital investigates red blood cell biology to develop new treatments for sickle cell disease and β-thalassemia, conditions affecting millions worldwide: https://www.lornegenome.org/mitch-weiss
Sickle cell and beta thalassemia - what are the treatment options and challenges? https://post.parliament.uk/research-briefings/post-pn-0754/
January 26, 2026 at 12:10 AM
Sickle cell and beta thalassemia - what are the treatment options and challenges? https://post.parliament.uk/research-briefings/post-pn-0754/
Are you planning on doing any more blood cells? I have a resident who I was thinking of giving an erythrocyte painting when she finishes, but she is very into hematology, so sickle cells or one with thalassemia would be cool.
January 23, 2026 at 8:16 PM
Are you planning on doing any more blood cells? I have a resident who I was thinking of giving an erythrocyte painting when she finishes, but she is very into hematology, so sickle cells or one with thalassemia would be cool.
Watching $VRTX; Vertex HDR CRISPR in HSCs undervalued - focus/exposure risk. analysi. https://www.biopharmadive.com/news/ASH-Vertex-Casgevy-childen-sickle-cell-thalassemia/807182/
January 22, 2026 at 2:06 PM
Watching $VRTX; Vertex HDR CRISPR in HSCs undervalued - focus/exposure risk. analysi. https://www.biopharmadive.com/news/ASH-Vertex-Casgevy-childen-sickle-cell-thalassemia/807182/
Found out I have Alpha thalassemia
Guess I'll always be sleepy an tired.
How fun
Guess I'll always be sleepy an tired.
How fun
January 22, 2026 at 4:55 AM
Found out I have Alpha thalassemia
Guess I'll always be sleepy an tired.
How fun
Guess I'll always be sleepy an tired.
How fun
In the United States most cases of Thalassemia occur in Americans of #Maltese, #Italian, #Greek, #Portuguese, or Levantine background.
January 18, 2026 at 1:42 PM
In the United States most cases of Thalassemia occur in Americans of #Maltese, #Italian, #Greek, #Portuguese, or Levantine background.
## Scalable Genome Editing Optimization via Adaptive Bayesian Reinforcement Learning for β-Globin Cluster Precision Correction in β-Thalassemia Intermedia
**Abstract:** β-Thalassemia Intermedia presents a significant therapeutic challenge due to variable disease severity and potential off-target…
**Abstract:** β-Thalassemia Intermedia presents a significant therapeutic challenge due to variable disease severity and potential off-target…
## Scalable Genome Editing Optimization via Adaptive Bayesian Reinforcement Learning for β-Globin Cluster Precision Correction in β-Thalassemia Intermedia
**Abstract:** β-Thalassemia Intermedia presents a significant therapeutic challenge due to variable disease severity and potential off-target effects of genome editing. Current CRISPR-based therapies often lack the precision and adaptive capacity required for personalized and safe treatment. This paper proposes a novel system, **Adaptive Genome Editing Optimization Network (AGEON)**, leveraging Adaptive Bayesian Reinforcement Learning (ABRL) to dynamically optimize CRISPR guide RNA (gRNA) selection and Cas enzyme parameters for precise β-globin cluster correction.
freederia.com
January 18, 2026 at 10:20 AM
## Scalable Genome Editing Optimization via Adaptive Bayesian Reinforcement Learning for β-Globin Cluster Precision Correction in β-Thalassemia Intermedia
**Abstract:** β-Thalassemia Intermedia presents a significant therapeutic challenge due to variable disease severity and potential off-target…
**Abstract:** β-Thalassemia Intermedia presents a significant therapeutic challenge due to variable disease severity and potential off-target…
## Automated CRISPR-Cas13a-Mediated Gene Editing for Targeted MicroRNA Modulation in β-Globin mRNA Splicing Correction for β-Thalassemia Minor
**Abstract:** This research proposes an automated, closed-loop CRISPR-Cas13a system for precisely modulating microRNA (miRNA) activity to correct aberrant…
**Abstract:** This research proposes an automated, closed-loop CRISPR-Cas13a system for precisely modulating microRNA (miRNA) activity to correct aberrant…
## Automated CRISPR-Cas13a-Mediated Gene Editing for Targeted MicroRNA Modulation in β-Globin mRNA Splicing Correction for β-Thalassemia Minor
**Abstract:** This research proposes an automated, closed-loop CRISPR-Cas13a system for precisely modulating microRNA (miRNA) activity to correct aberrant splicing of β-globin mRNA in patients with β-thalassemia minor. Utilizing a novel feedback mechanism integrating real-time transcriptomic analysis and automated guide RNA (gRNA) optimization, the system dynamically adjusts CRISPR-Cas13a expression and targeting specificity to achieve stable and efficient correction with minimal off-target effects.
freederia.com
January 18, 2026 at 6:52 AM
## Automated CRISPR-Cas13a-Mediated Gene Editing for Targeted MicroRNA Modulation in β-Globin mRNA Splicing Correction for β-Thalassemia Minor
**Abstract:** This research proposes an automated, closed-loop CRISPR-Cas13a system for precisely modulating microRNA (miRNA) activity to correct aberrant…
**Abstract:** This research proposes an automated, closed-loop CRISPR-Cas13a system for precisely modulating microRNA (miRNA) activity to correct aberrant…