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CURRENT INITIATIVES

Coordinating global DRPLA Natural History and Biomarker study anticipated to begin Fall, 2020. Details to be distributed through RareConnect DRPLA subgroup.

Premier New York hospital collecting skin biopsies from DRPLA patients worldwide and developing fibroblasts from those biopsies. Fibroblasts then to be reprogrammed into induced pluri-potent stem (“iPS”) cells to be tested against candidate ASOs and gene therapy.

Contracted with two researchers, one in the US and one in the UK, to develop and trial anti-sense oligonucleotides using DRPLA mice and iPS cells.

Contracted with one US biotech company to create a gene therapy to be trialed using DRPLA mice and iPS cells.

Partnered with Ataxia UK to create a DRPLA research initiative whereby CureDRPLA will leverage Ataxia UK’s depth of experience with ataxia research.

DRPLA patient registry in at least 4 languages to go live by Fall 2020.

CureDRPLA partnering with National Ataxia Foundation for virtual Externally Led – Patient Focused Drug Development (EL-PFDD) meeting on September 25, 2020.

CureDRPLA hired a US advisor Dr. Jeffrey Carroll. Dr Carroll is well known for his Huntington’s disease research and advocacy.

Activated the DRPLA RareConnect subgroup as a way to distribute new information.

Members from the CureDRPLA network attended, spoke at and/or exhibited at the following 2019 conferences:

  1. Global Genes RARE Drug Development Symposium 2019;
  2. Global Genes Patient Advocacy 2019;
  3. International Movement Disorders Society 2019;
  4. Manchester Rare Disease Showcase 20019
  5. Ataxia UK annual conference 2019; and
  6. International Ataxia Research Conference 2019.