Limb-Girdle Muscular Dystrophy

Watchlist

(log in to enable)

Retrieved

2022-04-26

Source

Gard

Trials

—

Genes

LMNA, SGCG, MYOT, DES, CAV3, FKRP, POMT2, DAG1, TTN, ANO5, SGCA, TRIM32, HNRNPDL, CRPPA, DYSF, DNAJB6, CAPN3, SGCD, SGCB, POMT1, TCAP, FKTN, POMGNT1, POMK, DPM3, PLEC, LIMS2, BVES, POMGNT2, GNE

LMNA, SGCG, MYOT, DES, CAV3, FKRP, POMT2, DAG1, TTN, ANO5, SGCA, TRIM32, HNRNPDL, CRPPA, DYSF, DNAJB6, CAPN3, SGCD, SGCB, POMT1, TCAP, FKTN, POMGNT1, POMK, DPM3, PLEC, LIMS2, BVES, POMGNT2, GNE, DOK7, RAPSN, LAMA2, EMD, TRAPPC11, ACTA1, SYNE2, ASTN2, TTN-AS1, SYNE1, DMD, TMEM43, FHL1, HNRNPA1, GMPPB, TNPO3, SSPN, COL6A2, COL6A3, TOR1AIP1, EIF3K, BEST1, FLNC, PYROXD1, SYVN1, POGLUT1, TRIM72, H19, SYTL2, PNO1, CMYA5, TARDBP, BRD4, SRRM2, MMD, DNAJB1P1, FBXO40, CBLL2, POPDC3, MUL1, SPATA18, MYOF, DCAF6, MAK16, RBMS3, RTEL1, OBSL1, AAVS1, SORBS1, CAP1, IL9, DNAJB1, HSPA4, HPS1, HNRNPD, HNRNPA2B1, MSTN, FXN, EGF, DCTN1, CTNS, CTAA1, COL6A1, CALD1, AQP4, AGT, ACTN2, ILK, LNPEP, MMP9, VCP, FST, BCAP31, ABCB6, RIPOR2, HACD1, HBHR, ALMS1, UTRN, PRKN, TGFB2, TBXT, SLC3A1, RAB27A, PYGM, PTBP1, SERPINB6, CFLAR

Drugs

505 amino acid protein, corresponding to amino acids 2-506 of the wild type human histidyl-tRNA synthetase, Adeno associated viral vector containing the human calpain 3 gene, Adeno-associated viral vector containing the human alpha-sarcoglycan gene

Interested in hearing about new therapies?

Limb-girdle muscular dystrophy is a group of disorders which affect the voluntary muscles around the hips and shoulders. The conditions are progressive, leading to a loss of muscle strength and bulk over a number of years. Onset may occur in childhood, adolescence, young adulthood, or even later. Males and females are affected in equal numbers. Most forms of limb girdle muscular dystrophy are inherited in an autosomal recessive manner. Several rare forms are inherited in an autosomal dominant pattern. While there are no treatments which directly reverse the muscle weakness associated with this condition, supportive treatment can decrease the complications. There are at least 20 different types of limb-girdle muscular dystrophy.