Genetic therapeutics researcher | Rare disease advocate | Sci-activist | Bengali SciFi writer | Overcaffeinated | Foodie | First-gen | 🇧🇩 ♡︎/⟳ ≠ endorsement
Palo Alto, CA
Joined April 2013
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DMD-Null mice (i.e., lacking all dystrophin isoforms) exhibit severe muscle weakness and poor regeneration. This proves that shorter dystrophin isoforms do aid regeneration to some extent.
pnas.org/doi/10.1073/pnas.26…
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Bottlebrush polymer that protects muscle membranes. Interesting development. pnas.org/doi/10.1073/pnas.25….
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Excited to attend the #ASGCTBreakthroughs24 conference by @ASGCTherapy and @MDAorg in Chicago! If you are around, please stop by my poster on improving antisense oligonucleotide efficacy with small-molecule enhancers.
📍 Poster #44
🗓️ Tuesday, Nov 17th, 5:45 PM
See you there!
This is true for more than just dysferlinopathies!
People with the same genetic disease often miss clinical trials because their symptoms/conditions have different names. A unified system based on genetic causes, NOT symptoms, would help more patients access trials & treatments.
Clinical trials for LGMD R2/LGMD 2B are currently underway, but patients with other Dysferlin gene mutations cannot participate. Therefore, it has attracted attention from LGMD R2/LGMD 2B patients, but no response from MMD/MM, DMAT, or DACM patients.
The reason is that because the disease names are categorized based on the phenotype, many people are unaware of the following:🥲
1. I don't know that it's a muscle disease that develops with the same causative gene
2. Not knowing that they can be treated together
3. There are cases in which even related Brothers and sisters have different disease names
*If the medical institution is different
Pharmaceutical companies will never conduct clinical trials on MMD/MM after LGMD R2/LGMD 2B. The reason for this is that it takes a lot of effort, time, and research expenses. Also, not everyone can take part in a clinical trial.
If things continue as they are, I think it is possible that some countries will not be able to recruit participants and clinical trials will not be able to be conducted, or that the trials will end prematurely. The reason for this is that although it is a rare disease, muscle diseases with the same causative gene are classified by phenotype.
I think this is somewhat of a tragic situation.😔💔
I hope that people with the same disease will seriously consider this issue.🌎🌍🌏
#MiyoshiMuscularDystrophy #MiyoshiMyopathy
#LGMDR2 #LGMD2B #DMAT #DACM
#Dysferlin
#Dysferlinopathy
#MuscularDystrophy
#WorldMuscleSociety
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It's been 4 days since I last spoke to my family back home. Telecommunication is completely shut down. A TV centre near my place was torched. The attackers keep on targeting innocent and unarmed #students all over #Bangladesh. God knows what's happening there.
So suffocating!
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Feeling helpless and ashamed.
Nearly 70 innocent souls lost, including minors, and hundreds injured.
Media & communication are heavily restricted, making it hard for the truth to come out. Can't contact my family and friends.
#Bangladesh #EndViolence
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No offense to CRISPR 2.0, but 'CRISPR 1.0' doesn't really 'hack through' DNA. Several clinical trials have shown 'CRISPR 1.0' to be quite precise. Misleading language undermines scientific progress. @GENbio, please try be a little more accurate in representing these advancements.
This quoted post is unavailable.
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This cyclone is so devastating!
It's strong but slow-paced, just now crossing my city with heavy rain & intense winds. It has caused the strongest and highest tide Bangladesh has seen since 1970.
#PrayForBangladesh #CycloneRemal
#CycloneRemal hit Sundarbans-Mongla in Bangladesh's Southwest this morning, <200 km from my place. Intense rain & winds have caused widespread damage. Many have lost everything. One of my acquaintances has had their house washed away.
If you are in the area & can help, please do!
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#CycloneRemal hit Sundarbans-Mongla in Bangladesh's Southwest this morning, <200 km from my place. Intense rain & winds have caused widespread damage. Many have lost everything. One of my acquaintances has had their house washed away.
If you are in the area & can help, please do!
Absolutely thrilled to share that I received an Outstanding Poster Award at #ASGCT2024 for my work on enhancing #ASO efficacy with small molecule enhancers! Grateful for the incredible support from my mentor, Dr. Yokota, and my amazing labmates.🔬 #Research #Science
Special thanks to my collaborators, Dr. Moulton and Dr. Bittner from the @OregonState, for their invaluable support. Most importantly, a huge thanks to everyone who stopped by my poster!
Last but not the least, thank you, @ASGCTherapy for this amazing opportunity!
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Amazing 5 days at #ASGCT2024!
Met wonderful scientists, enjoyed talks by legendary figures, and viewed awesome posters. Received great feedback on my poster and had insightful discussions on my work and others'. Learned so much and connected with many whose papers I've admired!
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My biggest takeaway from #ASGCT2024? I suppose in vivo gene modification technologies are not just a future possibility—they're becoming a transformative reality. So many promising programs and ongoing developments are set to truly expand their potential.
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This week, I'm at #ASGCT2024 in Baltimore, MD! If you're around, stop by and visit my poster on enhancing antisense oligonucleotide efficacy with small molecule enhancers. 🧬🔬
📍Poster Board #723
🗓️Wednesday, May 8th, 12:00 PM
Hope to see you there!
Big news: CASGEVY, the first CRISPR therapy, gets the green light for sickle cell and ß-thalassemia treatment!
#GeneticTherapy #CRISPR #Casgevy
investors.vrtx.com/news-rele…
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This is indeed a huge. Yet, we must confront the reality: having worked with hemoglobinopathies in Bangladesh, part of the so-called global 'thalassemia belt,' I understand that access to such breakthrough therapies still remains only a distant reality. #HealthInequity
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Excited to be featured by @WCHRI_UofA! We aim to develop a #treatment for facioscapulohumeral muscular dystrophy #FSHD, arguably the most complex genetic condition known. Thanks WCHRI, for backing our fight against complex and currently untreatable diseases. #GradSchool #Research
Saeed Anwar, WCHRI trainee, is working to treat muscular dystrophy with a DNA-like "weed killer." Saheed’s research could bring hope to those affected by this genetic condition.
Read more:
wchri.org/our-impact/stories…
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@WCHRIUofA
PS: my name is spelled 'Saeed,' not 'Saheed.' Nevertheless, I'm really grateful for the opportunity to share my work with you. 😊
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Encountering discrepancies in @GGGenome's search results for short seqs, e.g., no mismatches counted in mismatch-containing hits (& opposite). Contacted twice via web form @ gggenome.dbcls.jp with no reply in 3 wks. Seeking alternative communication to discuss the issue.
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So deeply saddened and shocked and concerned by the news. Prattay committed suicide just ten days ago, and now Avijit! Unbelievable!
I do not know what is happening and I cannot think anything! marquettewire.org/?p=4047943
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Check out our new meta-analysis article published in the @JPM_MDPI. We identified several exons that deemed promising targets of exon skipping therapy development.
doi.org/10.3390/jpm11010046
#DMD #ExonSkipping #GenotypePhenotypeAssociation #YokotaLab #Ualberta_FoMD
I'm pleased to share our latest research published in @PLOSONE! This was the first nation-wide survey on consanguinity in Bangladesh. It highlights the harmful consequences of consanguinity on reproductive behavior & the incidence of hereditary conditions.
doi.org/10.1371/journal.pone…
I'm pleased to share our latest research published in the Hemoglobin! For the first time in Bangladesh, we worked on α-thalassemia using umbilical cord blood samples and assessed the prevalence of different α-globin mutations!
doi.org/10.1080/03630269.202…
@tandfonline @Hemoglobin