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Mashup Score: 0
Dr. Steve Hughes, CMO at Avidity, explains the mechanism of action for AOC 1044 as a Duchenne muscular dystropy (DMD) treatment.
Source: CheckRare.comCategories: Hem/Onc News and Journals, Latest HeadlinesTweet
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Mashup Score: 0Mimicking sarcolemmal damage in vitro: a contractile 3D model of skeletal muscle for drug testing in Duchenne muscular dystrophy - IOPscience - 7 month(s) ago
This site uses cookies. By continuing to use this site you agree to our use of cookies. To find out more, see our Privacy and Cookies policy. Close this notification Ainoa Tejedera-Villafranca 1, Marisol Montolio 2,3, Javier Ramón-Azcón 5,1,4 and Juan M Fernández-Costa 5,1 •, Volume 15, Number 4 Citation Ainoa Tejedera-Villafranca et al 2023 Biofabrication 15 045024 DOI 10.1088/1758-5090/acfb3d 1 Institute for Bioengineering of Catalonia (IBEC), The Barcelona Institute of Science and Technology (BIST),
Source: iopscience.iop.orgCategories: General Medicine News, Latest HeadlinesTweet-
An artificial #muscle to study Duchenne #muscularDystrophy https://t.co/kNnEAQNGYJ https://t.co/sqfHMD5KLV
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Mashup Score: 0FDA Approves First Gene Therapy for Treatment of Certain Patients with Duchenne Muscular Dystrophy - 8 month(s) ago
FDA Approves First Gene Therapy for Treatment of Certain Patients with Duchenne Muscular Dystrophy
Source: www.fda.govCategories: Healthcare Professionals, Latest HeadlinesTweet-
.@US_FDA Approves First #GeneTherapy for Treatment of Certain Patients with #DuchenneMuscularDystrophy https://t.co/D8ZH00y0gU // #duchenne #musculardystrophy
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Mashup Score: 0Sarepta Therapeutics Announces FDA Accelerated Approval of Elevidys to Treat Children with Duchenne Muscular Dystrophy - Practical Neurology - 10 month(s) ago
Through its Accelerated Approval Pathway, the Food and Drug Administration (FDA) has approved Elevidys (delandistrogene moxeparvovec-rokl; Sarepta The
Source: Practical NeurologyCategories: Latest Headlines, NeurologyTweet-
https://t.co/hixKV30Gml @Sarepta #Duchenne #MuscularDystrophy #NeuroResearch #GeneTherapy
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Mashup Score: 1Wolters Kluwer Health - 10 month(s) ago
JavaScript Error JavaScript has been disabled on your browser. You must enable it to continue. Here’s how to enable JavaScript in the following browsers: Internet Explorer From the Tools menu, select Options Click the Content tab Select Enable JavaScript Firefox From the Tools…
Source: lww.comCategories: Latest Headlines, NeurologyTweet-
The @US_FDA has approved a new gene therapy from Sarepta Therapeutics, SRP-9001 (delandistrogene moxeparvovec), for Duchenne #MuscularDystrophy: https://t.co/bHYPsNVSq2 #neurotwitter #BreakingNews @AANMember https://t.co/qbpag55t21
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Mashup Score: 15Myoscaffolds reveal laminin scarring is detrimental for stem cell function while sarcospan induces compensatory fibrosis - npj Regenerative Medicine - 11 month(s) ago
We developed an on-slide decellularization approach to generate acellular extracellular matrix (ECM) myoscaffolds that can be repopulated with various cell types to interrogate cell-ECM interactions. Using this platform, we investigated whether fibrotic ECM scarring affected human skeletal muscle progenitor cell (SMPC) functions that are essential for myoregeneration. SMPCs exhibited robust…
Source: NatureCategories: General Medicine News, Latest HeadlinesTweet-
Study: Scarring of collagen 'highway' prevents #stemcells from healing damaged tissue in Duchenne #musculardystrophy https://t.co/ibf4Lusf4G https://t.co/qWa5AVISZO
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Mashup Score: 1The lesson of Terry, the pioneer patient who didn’t make it - 12 month(s) ago
Terry and Richard Horgan (Courtesy of Cure Rare Disease) He was the first patient to get a CRISPR therapy for muscular dystrophy. The first to receive a CRISPR treatment made specifically for him. …
Source: CRISPeR FRENZYCategories: Future of Medicine, Latest HeadlinesTweet-
Terry Horgan, the first patient to receive an epigenetic #CRISPR therapy for muscular dystrophy, passed away due to an adverse reaction to the viral vectors used to deliver the therapy, not the epigenetic correction made. #musculardystrophy https://t.co/FRFOdkMGSD
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Mashup Score: 8Defective BVES-mediated feedback control of cAMP in muscular dystrophy - Nature Communications - 1 year(s) ago
cAMP is an important second messenger involved in many aspects of biological processes. Here, Li et al. shows that blood vessel epicardial substance (BVES) is a negative regulator of cAMP signaling required for maintaining muscle mass and function.
Source: NatureCategories: General Medicine News, Latest HeadlinesTweet-
Researchers investigate the protein BVES and its important role in #musculardystrophy @iumedschool @NatureComms https://t.co/vRIcxOhs64 https://t.co/Xtym6Lv8Sx
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Mashup Score: 3TRAPPC2L-related disorder: first homozygous protein-truncating variant and further delineation of the phenotype - 1 year(s) ago
The TRAPP (TRAfficking Protein Particle) complexes are evolutionarily conserved tethering factors involved in the intracellular transport of vesicles for secretion and autophagy processes. Pathogenic variants in 8 genes (of 14) encoding TRAPP proteins are involved in ultra-rare human diseases, called TRAPPopathies. Seven of them are autosomal recessive neurodevelopmental disorders with…
Source: Journal of Medical GeneticsCategories: General Medicine Journals and Societies, Latest HeadlinesTweet-
#TRAPPC2L syndrome: a severe neurodevelopmental disorder and a rare congenital #MuscularDystrophy First homozygous protein-truncating variant & further phenotype delineation ➡️ https://t.co/hz70mVcLsq @mario_obj @FlorenceRicc Image #Alphafold via @uniprot https://t.co/Cns6SisfXo
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Mashup Score: 0Sysnav Healthcare announces a multi-year collaboration with Roche based on Sysnav's proprietary movement tracking technology - 1 year(s) ago
/PRNewswire/ — Sysnav Healthcare and Roche enter a partnership to facilitate development of industry standard outcome measurements in diseases associated with…
Source: www.prnewswire.comCategories: Healthcare Professionals, Latest HeadlinesTweet-
Sysnav Healthcare announces a multi-year collaboration with @Roche based on Sysnav's proprietary #movement tracking technology | #musculardystrophy #wearables https://t.co/o09X5UzVyj
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Steve Hughes, MD, chief medical officer at Avidity Biosciences, explains the mechanism of action for AOC 1044 as a Duchenne muscular dystrophy #DMD treatment #checkrare #musculardystrophy https://t.co/1fLC3TP1bC