Sverige för att ge patienter snabb tillgång till nya läkemedel och metoder

Under utveckling finns också genterapi. Det innebär att man i celler, som har en felaktig gensekvens, för in DNA-sekvenser med frisk information, som tar över den sjukliga processen och därmed kan man med engångsinsats förändra och förhoppningsvis bota patienten.

Och Sverige ska satsa på de nya möjligheter som cell- och genterapi kan ge – kring den slutsatsen fanns en stor enighet vid ett seminarium i Almedalen arrangerat av LIF och Dagens Nyheter.

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Neuroförbundet

New Study Reports: Nusinersen raises SMN Protein Levels

Exicure to Present Data at the CureSMA Annual Conference in Dallas

SKOKIE, Ill.–(BUSINESS WIRE)–Exicure, Inc. (OTCQB:XCUR), the pioneer in gene regulatory and immunotherapeutic drugs utilizing three-dimensional, spherical nucleic acid (SNA™) constructs, announced today that Exicure and its collaborators at The Ohio State University Wexner Medical Center will show preclinical data demonstrating the performance of Exicure’s SNA compound designed for use in spinal muscular atrophy (SMA). These data will be presented at the Cure SMA Annual Conference in Dallas, Texas on June 14, 2018.

“Exicure’s spherical nucleic acid version of nusinersen demonstrates increased survival and decreased toxicity in the translationally-relevant SMA mouse model,” said David Giljohann, PhD, CEO of Exicure. “We believe these results are important for developing improved treatments for patients with SMA. These data also suggest that Exicure’s technology platform could potentially create more potent therapies for other disorders of the central nervous system, including Huntington’s disease, Alzheimer’s disease, and Parkinson’s disease.”

At the meeting, Arthur Burghes, PhD, from Ohio State’s Wexner Medical Center, and Exicure will present data from preclinical studies in a SMA mouse model. The poster is titled “Nusinersen in spherical nucleic acid (SNA) format improves efficacy both in vitro in SMA patient fibroblasts and in Δ7 SMA mice and reduces toxicity in mice.” The presentation will highlight that Exicure’s proprietary technology:

  • Prolonged survival by four-fold (maximal survival of 115 days compared to 28 days for nusinersen-treated mice);
  • Doubled the levels of healthy full-length SMN2 mRNA and protein in SMA patient fibroblasts when compared to nusinersen;
  • Doubled the quantity of healthy full-length SMN mRNA levels in spinal cord tissue compared to untreated mice;
  • Mitigated toxicity of nusinersen at the highest dose tested in mice.

In August 2017, Exicure and The Ohio State University established a collaboration to further validate and characterize the pharmacology of Exicure’s nusinersen-SNA compound in mouse models. This collaboration’s ongoing in vivo research is conducted by Dr. Burghes, an internationally known researcher, leading basic and clinical research on SMA and other genetic neuromuscular disorders.

En sällsynt resa

Anna Hallgren är socionom och sexualterapeut och lever med den sällsynta muskelsjukdomen nemalinmyopati. Här delar hon tillsammans med sin dotter och sin mamma Ingalill med sig av sina erfarenheter av att leva med sjukdomen. De pratar också om hur vårdens kunskap om och bemötande av personer med sällsynta diagnoser kan förbättras. Inspelat den 28 februari 2018 på Folkets hus i Göteborg.

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Sällsynta dagen 2018