Rezultati pretraživanja
  1. 5. kol 2019.

    Great visit with Sarepta. Looking forward to catching up with the team again at the LGMD conference.

  2. 3. lis 2019.
    Odgovor korisniku/ci

    As a member of the community, thanks for the good wishes! I'm optimistic, but we'll see.

  3. 8. sij

    Collaborative effort with . . . Inhibition of DNAJ-HSP70 interaction improves strength in muscular dystrophy

  4. 6. lis 2019.

    I made a short video giving my thoughts about the 2E gene therapy trial and the data released on Friday.

  5. Breaking News: has announced positive functional data from its trial testing SRP-9003, a gene therapy for 2E. Find out more >>

    Breaking News Research graphic.png
  6. 26. velj 2019.

    Since we’re a cult, while we wait for data... Last light over the Rockies tonight..... I’m taking that as a sign from above for Community & step forward for rare disease patients everywhere....

  7. 26. kol 2019.

    Sarepta latest conference. This time LGMD

  8. 14. stu 2019.

    All the criticism about not focused anymore, are completely wrong, it's a future rights deal, to get it before someone else do, or pay x100 later in 2-3 years as paid $8.7B for & paid $165M for Myonexus's !

    Prikaži ovu nit
  9. 27. pro 2018.

    secret ingredient to produce phenomenal results on Micro D patient #4. Not disclosed. Probability of transferable to ? I assume yes dt numerous similarities.

  10. A new global study, funded by , is looking for participants with LGMD2B/R2 or Miyoshi myopathy to help move research forward at . Read here how to get involved >>

    Research lab
  11. 9. ruj 2019.
    Odgovor korisnicima

    Hi! I’m a 4th year PhD student studying and I have (limb girdle muscular dystrophy)

  12. 10. ruj 2019.

    patient newsletter: Pg 1: Patient affairs attended events Pg 2: Tamara, new 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)

  13. 7. lis 2019.
    Odgovor korisniku/ci

    In 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.

  14. 5. lis 2019.

    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

  15. 9. svi 2019.

    Our new paper is now out in : First high throughput screening for Limb-girdle muscular dystrophy type 2D (LGMD2D). Great collaborative work between and cc cc

  16. prije 15 sati

    Last night was rough. I caught a cold, and b/c of a weak cough (), 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.

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  17. 7. lis 2019.

    An uplifting article to start the week off with. Nicole Robertson writes an open article about living with . Original post on

  18. 10. ožu 2019.

    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 and the first national conference dedicated to treating this disease

  19. 3. lip 2019.

    Giving a big well done shout out this to Julie & Jean who completed the yesterday in support of for 3 year old Matilda who has

  20. 15. sij

    Nothing tops human genetics when understanding protein function. Mutations in the J domain of DNAJB6 cause dominant distal myopathy

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