Discover and read the best of Twitter Threads about #SCD

Most recents (24)

1) Welcome to Part 2 of a new #accredited #tweetorial in our series of educational programs on #hypertrophic #cardiomyopathy #HCM. DOn't miss Part 1, still available for 🆓CE/#CME, at cardiometabolic-ce.com/hcm5a/.
Now you can learn more, and EARN MORE credit, by following this 🧵!
2) Our expert author is again Sergio Kaiser MD PhD FACC FESC 🇧🇷🇮🇱 @pabeda1, cardiologist 🫀, Professor 🎓 of #InternalMedicine, Rio de Janeiro State University. He brings the general cardiologist's perspective to our #HCM discussions. Read and learn!
#FOAMed #CardioTwitter Image
3) This program is supported by an unrestricted educational grant from Bristol Myers Squibb. Statement of accreditation and faculty disclosures at cardiometabolic-ce.com/disclosures/. Credit for #physicians #nursepractitioners #physicianassociates #nurses #pharmacists from @academiccme.
Read 62 tweets
1) Welcome to Part 1 of a new #accredited #tweetorial in our series of educational programs on #hypertrophic #cardiomyopathy #HCM. Previous programs, still available for 🆓CE/#CME, are at cardiometabolic-ce.com/category/hcm/.
Now you can earn another 1.5hr credit by following ALL of this 🧵!
2) Our expert author is Sergio Kaiser MD PhD FACC FESC 🇧🇷🇮🇱 @pabeda1, cardiologist 🫀, Professor 🎓 of #InternalMedicine, Rio de Janeiro State University. He brings the general cardiologist's perspective to our #HCM discussions. Read and learn!
#FOAMed #CardioTwitter
3) This program is supported by an unrestricted educational grant from Bristol Myers Squibb. Statement of accreditation and faculty disclosures at cardiometabolic-ce.com/disclosures/. Credit for #physicians #nursepractitioners #physicianassociates #nurses #pharmacists from @academiccme.
Read 68 tweets
1) Welcome to a new #accredited #tweetorial in our series of educational programs on #hypertrophic #cardiomyopathy #HCM. Previous programs, still available for 🆓CE/#CME, are at cardiometabolic-ce.com/category/hcm/.
Now you can earn another 0.75hr credit by following this 🧵!
2) Our expert author is JA Linderbaum MS, ARNP, FACC, FPCNA @jlinderbaum, Associate Professor of Medicine, @MayoClinic, CV #NursePractitioner, Assoc. Medical Editor #AskMayoExpert.
#FOAMed #MedEd @MedTweetorials #CardioTwitter @transformingHC @TNPJ_Journal #cardiology
3) This program is supported by an unrestricted educational grant from Bristol Myers-Squibb. Statement of accreditation and faculty disclosures at cardiometabolic-ce.com/disclosures/. Credit for #physicians #nursepractitioners #physicianassociates #nurses #pharmacists from @academiccme.
Read 70 tweets
1/As promised & after reading $CRSP latest Q4 2022 #financial #report here is my impression regarding @CRISPRTX latest corporate status. I have focused only on the main issues that I found to be the most interesting & relevant. #CRISPR #BioTech #FinTwit #Genomics #GeneEditing 🧵 CRISPR Therapeutics, a biopharmaceutical company focused on
2/IMO the most significant corporate event in Q4 was $CRSP announcement that regulatory Exa-cel submissions of both #SickleCell & #BetaThalassemia validated in the #EU & #UK & that the @US_FDA BLA submission is on track by the end of Q1 ‘23 - possibly reaching the #markets in ‘23 Regulatory submissions complete for exagamglogene autotemcel
3/Exa-cel/CTX001 - the key program in $CRSP portfolio, is an #autologous Ex-Vivo #CRISPR/#Cas9 #GeneEditing therapy aimed for patients suffering from #TDT or severe #SCD. The latest readout for both programs was phenomenal with 42/44 TDT & 31/31(!) demonstrated remarkable results 3/Exa-cel - formally known as CTX001 and the key program in
Read 17 tweets
1/@GraphiteBio announced that after reviewing its business, it decided to discontinue any further development of nulabeglogene autogedtemcel (nula-cel) for #SickleCell disease, to explore a range of strategic alternatives & to reduce 50% in its workforce! #CRISPR #BioTech $GRPH Graphite Bio today announce...
2/@GraphiteBio is also exploring the potential to continue nula-cel development externally. $GRPH intends to continue research activities associated with its early-stage non- genotoxic conditioning program, with the goal of advancing toward potential development candidates.👇
3/In addition, @GraphiteBio’s Board of Directors has approved a corporate restructuring that would reduce the company’s workforce by approximately 50%, among other actions to reduce cash burn while the company explores strategic alternatives. $GRPH Image
Read 5 tweets
1) Welcome to a new #accredited #tweetorial on #hypertrophic #cardiomyopathy #HCM. Our expert author is Ahmad Masri MD @MasriAhmadMD of @OHSUCardio where he leads the Hypertrophic Cardiomyopathy Ctr. See a previous program from him, on #Fabry disease, at cardiometabolic-ce.com/cardiorenal-fa…. Image
2a) This program is intended for #healthcare providers and is supported by an educational grant from Bristol-Myers Squibb. Statement of accreditation and faculty disclosures at cardiometabolic-ce.com/disclosures/. FOLLOW US for the most timely expert education in #cardiometabolic care!
2b) #Physicians #Nurses #PhysicianAssociates #NursePractitioners #Pharmacists earn a full 1⃣ hour of 🆓CE/#CME credit from following this 🧵. For educational purposes, Twitter still works!! 😁
Read 93 tweets
How many patients are on disease modifying treatments for #SCD - data from 2014-2021 presented by Dr. Terri Newman. @ash22 1/n
We're talking about #Hydroxyurea and in 2017, expanded use of #HU as well as L-glutamine and then in 2019 #voxelotor and #crizanlizumab. #ASH22 2/n
They looked at 5022 patients with Rx of DMTs. Those on DMTs were surprisingly mostly in the 18-45 group (the box is wrong on the screenshot) and mostly male and had more VOCs. #ASH22 3/n Image
Read 5 tweets
Management of pregnant women with #SCD in high income countries with Dr. Eugene Oteng-Ntim (with the cutest intro from Dr. Eugenia Asare). Writing down the #pearls here so I can share with my patients for their future and so I can advocate for them. #ASH22 1/n
As we know, more low/not detectable AMH levels in women with #SCD. Know that preimplantation diagnosis and successful transfer is possible with data to support that. And many want #NIPT secondary to fear of amnio or CVS, which can be done. (w/ @Nonacus) #ASH22 2/n
What risks are we talking about in sickle cell disease and pregnancy? 1/4 of the patients in their cohort required transfusion during pregnancy and RR of preeclampsia is 2.43 and 3x risk of SGA, 4x risk of stillbirth. #ASH22 3/n
Read 5 tweets
1/@GraphiteBio presented preclinical results supporting the use of a single-#cell #RNA #sequencing method to assess #gene correction outcomes in #patients treated with nulabeglogene autogedtemcel (nula-cel) - GPH-101. #BioTech #CRISPR #GeneEditing #Genomics $GRPH #ASH22 GraphiteBio presented precl...
2/GPH101 (nula-cel) is a #CRISPR #Cas9 #GeneEditing autologous #stem #cell-based #therapy in clinical development aimed to treat #SickleCellDisease. GPH101 is designed to directly correct the underlying mutation, thereby decreasing HbS production & restoring HbA expression #ASH22 GPH101 (nula-cel) is a CRIS...
3 $GRPH gene correction platform involves editing hematopoietic #stem cells found in the #bonemarrow that develop into various types of #blood #cells. Since red blood cells lose their genomic DNA during maturation-tracking #GeneEditing in mature cells via sequencing is impossible Graphite bio’s gene correct...
Read 7 tweets
#Voxelotor #SCD real world outcomes Q&A -- Q: what about concurrent HU vs not? A: we have thatdata, will get to it! 1/n Image
Q: what about stroke? A: it's mostly in pediatrics, and dataset is limited so REALLY important, but not from this data just yet. Here are his conclusions. 2/n Image
Q: improved outcomes here especially with VOC.. can we normalize against # of visits since pandemic may impact this? A: great point! Q: What about decreased ACS? A: No we haven't analyzed ACS but we have to have a # of rates to analyze it. 3/n
Read 4 tweets
Will be jumping around this afternoon -- but starting off with "Hormonal Contraceptive Use and Association with Thromboembolism in Women with Sickle Cell Disease" with Dr. Natasha Bala. I feel like this is gonna be a good one! Tune in below. #ASH22 1/n
Combined OCPs is a category 2 -- meaning the benefit outweighs the risks. Their hypothesis was that risk of thrombosis was higher with combined OCPs versus progesterone only. #ASH22 2/n
They excluded patients without Rx 6 months prior to include new hormonal agent patients only (and excluded ones that had a hx of VTE prior). Their definitions here. #ASH22 3/n
Read 8 tweets
Dr. Alan Anderson here to present #crizanluzumab (which is approved for age >16 to prevent VOE) and its effect on #priapism. Long prescreening to capture these occ events. Needed 4 in prescreening 14 weeks. 1/n #ASH22 Image
24 patients met the eligibility criteria. Received 5 mg/kg and then q4 weeks and checked outcome at week 26 and treated for 52 weeks. Also reported #PRO. #ASH22 2/n
This is the baseline data -- first reported at age 13-17 years, often daily or every other day. If this is surprising to you, think about how you are asking your #SCD pts about #priapism? It has a significant impact on their life! #ASH22 3/n Image
Read 6 tweets
[Rappel] Décret n° 2021-1572 du 3/12/2021 relatif au respect des exigences de l'intégrité scientifique par les établissements publics contribuant au service public de la recherche ... #SCD #BU #universités legifrance.gouv.fr/jorf/id/JORFTE…
1/6
Les établissements publics et fondations reconnues d'utilité publique ...
1° Assurent la sensibilisation et la formation de leurs personnels au respect des exigences de l'intégrité scientifique, ainsi que de leurs étudiants dans le cadre de la formation à et par la recherche 2/6
(...) 3° Promeuvent la diffusion des publications en accès ouvert et la mise à disposition des méthodes et protocoles, des données et des codes sources associés aux résultats de la recherche afin d'en garantir la traçabilité et la reproductibilité 3/6
Read 6 tweets
$SGMO status going into C22
Two synthetic gene transfer programs:
- HemA now run by $PFE P3. Hold for protocol amendment. Expect timing update for full analysis in next couple months.
- #Fabry owned program good data potential first/best in class
No further programs.
1/
$SGMO development focus leveraging ZF technology shows evolution from old four platforms to new integrated tech message. ZF tech facilitates both ex vivo and in vivo genomic medicines. Examples are CAR Tregs cell therapy and ZFP-TF CNS regulation.
2/ ImageImageImageImage
Cell therapy pipeline:
-Sickle cell collab with $SNY lead indication (see next)
-$GILD collab programs on hold. No update for a while but likely tied to durability issues seen at $ALLO $CRSP $DTIL
-Steadfast study is #POC for CAR Tregs with significant pipeline to follow
3/ ImageImageImageImage
Read 9 tweets
💃🕺Who's excited for tonight's #strictlyfinal? Keep updated with our live blog below or follow the latest via @mkmswain #strictly #strictly2021 #scd #scd2021 #strictlycomedancing @bbcstrictly
telegraph.co.uk/tv/2021/12/18/…
@RoseAylingEllis and @pernicegiovann1 and #JohnWaite and @jojo_radebe are tied after the first dance of the #strictlyfinal 💃🕺

Follow the latest on our live blog and via @mkmswain #strictly #strictly2021 #scd #scd2021 #strictlycomedancing

telegraph.co.uk/tv/2021/12/18/… Image
@RoseAylingEllis @pernicegiovann1 @jojo_radebe @mkmswain Our two #strictly2021 finalists are tied - with even @CraigRevHorwood cracking and giving both @RoseAylingEllis and #JohnWhaite a perfect score #strictlyfinal #strictly #strictlycomedancing #scd

Keep updated via our live blog and @mkmswain's tweets 💃🕺

telegraph.co.uk/tv/2021/12/18/… Image
Read 4 tweets
Gross incompetence on behalf of BBC?

Bad legal advice?

Surely it wasn't impossible to make full vaccination a condition of employment eg on H&S grounds?

"Strictly chaos as 'third professional dancer refuses vaccine'" metro.co.uk/2021/09/21/str… via @MetroUK
@MetroUK who are the three vaccine refuseniks?

If the BBC won't replace them now, the public should know, so we can vote them off ASAP.
@MetroUK is there anything that would prevent it being a condition of employment that Strictly Come Dancing #SCD competitors are fully vaccinated?
@AdamWagner1 @BarristerSecret @davidallengreen
Read 4 tweets
#ESCCongress #arrhythmia

Prediction of #suddencardiacdeath:

@BehrElijah on pathogenesis and risk stratification of #SCD in Brugada syndrome

1/
#ESCCongress #arrhythmia

Prediction of #suddencardiacdeath:

@BehrElijah on pathogenesis and risk stratification of #SCD in Brugada syndrome

2/
#ESCCongress #arrhythmia

Prediction of #suddencardiacdeath:

@BehrElijah on pathogenesis and risk stratification of #SCD in Brugada syndrome

3/
Read 4 tweets
#ESCCongress #arrhythmia

Prediction of #suddencardiacdeath:

K. Nakajima: Differential prediction of #SCD and HF death using machine learning and nuclear cardiology

2/
#ESCCongress #arrhythmia

Prediction of #suddencardiacdeath:

K. Nakajima: Differential prediction of #SCD and HF death using machine learning and nuclear cardiology

3/
Read 6 tweets
$SNY looks like they are ready for a Genomic Medicine Unity #GMU coming out year in C21. Many hires. Many open reqs. Building on Ablynx, Bioverativ and Kiadis M&A
#GeneTherapy
1/
$SNY #GMU head of Rare and Neurologic Disease Research is Christian Mueller. Video into is pretty good
Addl heads include Bruno Figueroa (CMC) and Cate O'Riordan (translational unit)

2/
$SNY #GMU Principal Scientist Robert Jackson profile says he is working on: Huntingtin's, MLD, PKU, Neurophathies, T1Diabetes Hypertension, Nephrophaty, vascular disease, Rheumatoid Arthritis and sensorineural hearing loss
3/
Read 14 tweets
When we chartered @theNASEM to provide a #SCD Strategic Plan & Blueprint for Action I hoped for a transformational doc for the next decade–and that is exactly what they provided. Good news– we have already started the journey but we still have a ways to go bit.ly/2FtAgdF
Today, @theNASEM released a new report: Addressing Sickle Cell Disease – A strategic Plan and Blueprint for Action.

This report outlines actionable strategies for addressing the care and treatment of people with #SCD.

Read now: bit.ly/2Fi1qVh Image
This @theNASEM report, charted by OASH & @MinorityHealth, makes recommendations on transitions from pediatric to adult #SCD care, research innovations, particularly gene-editing approaches, & the role of patient groups & community-based orgs.

Learn more: bit.ly/3himvMq
Read 3 tweets
This #WorldSickleCellDay, let us renew our commitment to improving the lives of everyone living with #SickleCell disease around the world and celebrate the important progress we’re making against the disease. Join the movement to help raise awareness about #SCD!
#SickleCellDisease is a serious health condition which affects the body’s red blood cells and can cause intense episodes of pain. Learn more about #SCD from @CDC_NCBDDD and @CDCgov: cdc.gov/ncbddd/sicklec…
As a pediatric intensive specialist I have cared for hundreds of children w/#SickleCell. They are my inspiration to fight every day. My goal & passion is to create a future where we can provide individuals w/#SCD a long life free from pain & complications. The time is now #WSCD20 Image
Read 5 tweets
Our goal at @HHSGov is to lead Americans to healthier lives and to provide a road map to a healthier nation. As such, we are focused on health innovation, transformation, and addressing emerging diseases like #Coronavirus. #AllHealthLive Image
@HHSGov @cspan There is no silver bullet when it comes to addressing the #OpioidEpidemic. All programs and offices must continue to work together across the country, especially as we work to combat the 4th wave of America’s overdose epidemic—methamphetamines. #AllHealthLive Image
@HHSGov @cspan At @HHSGov, we are also committed to extending the lives of Americans with sickle cell disease by at least 10 years, within 10 years. We are dedicated to improving the quality of life of those living with #SCD. Visit @MinorityHealth to learn more: minorityhealth.hhs.gov/sicklecell
Read 3 tweets
With @NIH leadership this morning discussing the importance of charting a course to stop the spread of viral #hepatitis and save lives. Image
I made the commitment to settle for no less than a plan that aims to eliminate viral hepatitis in our nation. We have the diagnostic tests, several effective therapies, and vaccines. Let’s make viral #hepatitis something only to read about in history books!
Also discussed a topic very dear to me, Sickle Cell Disease in Sub-Saharan Africa. So much progress has been made but more work needs to be done. We need to partnerships and collaboration to ensure the needs are reflected and adapted at the continent level. #SCD Image
Read 3 tweets
Today I’m in Orlando with @ASH_hematology meeting with Ghana, Uganda and Tanzania on #SickleCell disease. #SCD Image
@ASH_hematology 300,000 children are born with #SCD each year in Africa; most will die unnecessarily before age 5 years. Today, we continue our work to save this children with our partners @ASH_hematology and several African delegations.
@ASH_hematology As we work to improve the care of children with #SCD in Africa, we must re-double our efforts to improve care for SCD patients in our nation. Far too few patients receive standard of care, or appropriate pain prevention and management.
Read 3 tweets

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