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Great visit with Sarepta. Looking forward to catching up with the team again at the LGMD conference.
#srpt#lgmd https://twitter.com/Sarepta/status/1158439642921934848 …
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As a member of the
#LGMD community, thanks for the good wishes! I'm optimistic, but we'll see. -
Collaborative effort with
@GestwickiLab.#lgmd.#dnajb6. Inhibition of DNAJ-HSP70 interaction improves strength in muscular dystrophyhttps://www.biorxiv.org/content/10.1101/2020.01.03.893149v1 … -
I made a short video giving my thoughts about the
#LGMD 2E gene therapy trial and the data released on Friday. https://www.youtube.com/watch?v=UUeIcfAUXqM … -
Breaking News:
@Sarepta has announced positive functional data from its trial testing SRP-9003, a gene therapy for#LGMD 2E. Find out more >> http://bit.ly/2BmYBwX pic.twitter.com/AjaGsUQLII
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Since we’re a cult, while we wait for
$SRPT data... Last light over the Rockies tonight..... I’m taking that as a sign from above for#LGMD Community & step forward for rare disease patients everywhere.... pic.twitter.com/Dmxgjpjn6d
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$SRPT Sarepta latest conference. This time LGMD#LGMD https://twitter.com/GantosJ/status/1166067717742485504 …
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A new global study, funded by
@JainFoundation, is looking for participants with LGMD2B/R2 or Miyoshi myopathy to help move research forward at@jwmdrc. Read here how to get involved >> http://bit.ly/36sLK96#MusclesMatter#LGMD pic.twitter.com/dKuXpzpIto
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Hi! I’m a 4th year PhD student studying
#Biostatistics and I have#LGMD (limb girdle muscular dystrophy) -
$SRPT@Sarepta patient newsletter: Pg 1: Patient affairs attended events Pg 2: Tamara, new#LGMD patient affairs manager Kate, returning summer intern (worked LGMD Annual Meeting) Pg 3: Trial details 5051-201 still enrolling in FL & GA (PPMO x51 Ph2) http://join.parentprojectmd.org/site/DocServer/Sarepta_Patient_Affairs_Newsletter_Summer_2019.pdf?docID=19060&_ga=2.157400826.199558837.1568174979-1586938448.1568174979 … -
In
#LGMD NH studies, there can be *some* apparent improvement, usually because of person being sick/tired at first time point, or different evaluator. See slide 25 of talk: sustained improvement at multiple time points, also multiple patients/outcome measures. This seems real. -
$SRPT#LGMD@BradWil20444770 is the best source for anything LGMD. That’s his world. Go through his tweets for links to papers and insider perspectives if you’re wanting to learn about LGMD. He was a moderator at the LGMD annual meeting & Director of Research at@JainFoundationhttps://twitter.com/BradWil20444770/status/1180161745387524096 … -
Our new paper is now out in
@SciReports : First high throughput screening for Limb-girdle muscular dystrophy type 2D (LGMD2D). Great collaborative work between@GenethonFr and@IStemFrance#screening#LGMD#Inserm#genopole cc@BiotherapiesIns cc@GiLGMDhttps://www.nature.com/articles/s41598-019-43399-w … -
Last night was rough. I caught a cold, and b/c of a weak cough (
#lgmd), I spent the majority of the night coughing and praying to fall asleep. I know this is a quick burn in an able-bodied person b/c my hubby got it last week and was basically done in a day. This is day 3 for me.Prikaži ovu nit -
An uplifting article to start the week off with. Nicole Robertson writes an open article about living with
#lgmd#musculardystrophy. Original post on@TheMightySitehttps://www.yahoo.com/lifestyle/facing-challenges-life-limb-girdle-050420637.html … -
$SRPT National Limb Girdle Muscular Dystrophy Conference Labor Day weekend in Chicago August 30th to September 1st, 2019 * Largest gathering ever of patients and families with#LGMD and the first national conference dedicated to treating this disease …https://nationallimbgirdlemusculardystrophyconference.com/sponsorship?fbclid=IwAR1A0ClW8U-IjiFqzLarYL2IqjA8r4Mg78eovJB3Ln99YY4Ry_jv0iZ9Nfc … -
Giving a big well done shout out this
#MuscleMonday to Julie & Jean who completed the#Aberdeen@thekiltwalk yesterday in support of@MDUK_News#makingadifference for 3 year old#Granddaughter Matilda who has#LGMD#EveryDayCounts http://www.justgiving.com/fundraising/jools5 …pic.twitter.com/bYQ8PRYoLb
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Nothing tops human genetics when understanding protein function.
#myotwitter#lgmd Mutations in the J domain of DNAJB6 cause dominant distal myopathy https://www.nmd-journal.com/article/S0960-8966(19)31201-5/fulltext#.Xh8Y2j-azv4.twitter …
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