IgG kappa paraproteinaemia was discovered in one patient with amyotrophic lateral sclerosis and one with chronic spinal muscular atrophy. Serum from these patients was injected into mouse muscles paralysed by botulinum toxin which is known to induce sprouting from motor nerve terminals. Daily injection of serum for 7 days failed to inhibit terminal sprouting. It is concluded that the paraprotei...

Duchenne muscular dystrophy is a lethal X-linked recessive disorder caused by mutations in the dystrophin gene. Delivery of functionally effective levels of dystrophin to immunocompetent, adult mdx (dystrophin-deficient) mice has been challenging because of the size of the gene, immune responses against viral vectors, and inefficient infection of mature muscle. Here we show that high titer stoc...

Case Description- A five-year-old Arabian stallion weighing approximately 300 kg with large infected chronic torn wound on the neck and pectoral region 5 days after IM injection of flunixin meglumine with history of anorexia, depression and lack of response to antibacterial treatment referred to the Veterinary Teaching Hospital of Lorestan University. Clinical Fin...

Duchenne muscular dystrophy is an X-linked muscle disease characterized by mutations in the dystrophin gene. Many of these can be corrected at the posttranscriptional level by skipping the mutated exon. We have obtained persistent exon skipping in mdx mice by tail vein injection with an adeno-associated viral (AAV) vector expressing antisense sequences as part of the stable cellular U1 small nu...

The study assessed a radiolabeled depsipeptide conjugate (68Ga-DOTA-TBIA101) for its potential as an imaging agent targeting infection or infection-associated inflammation. 68Ga-labeled DOTA-TBIA101 imaging was performed in (NZR1) healthy rabbits; (NZR2) rabbits bearing muscular sterile inflammation and Staphylococcus aureus (SA) infection; and (NZR3) rabbits infected with Mycobacterium tubercu...

Two years after the Round Table of January 2004 which concluded with the possibility of utilizing exon skipping as a therapeutic strategy, the clinical potential of this strategy has been confirmed and has been translated into different research approaches (molecular, pharmaco-chemical, gene therapy, autologous cell therapy) which have all attained pre-clinical status today. Several clinical tr...

Antisense oligonucleotides (AOs) have the potential to induce functional dystrophin protein expression via exon skipping by restoring in-frame transcripts in the majority of patients suffering from Duchenne muscular dystrophy (DMD). AOs of morpholino phosphoroamidate (PMO) and 2'-O-methyl phosphorothioate RNA (2'Ome RNA) chemistry have been shown to restore dystrophin expression in skeletal mus...

objective a 4-month-old female with osteogenesis imperfecta (oi) type ii was admitted in picu of our center due to severe respiratory distress and fever with a diagnosis of severe pneumonia, and mechanical ventilation was initiated. due to severe hypotonia, ncv and emg were performed, and spinal muscular atrophy (sma) type i was diagnosed.

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