{"id":386,"date":"2024-08-19T23:39:30","date_gmt":"2024-08-19T23:39:30","guid":{"rendered":"https:\/\/simpathic.eu\/?page_id=386"},"modified":"2024-08-19T23:42:49","modified_gmt":"2024-08-19T23:42:49","slug":"read-more-focus-on-several-diseases","status":"publish","type":"page","link":"https:\/\/simpathic.eu\/?page_id=386","title":{"rendered":"Read more Focus on several diseases"},"content":{"rendered":"\t\t
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SIMPATHIC
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Focus on nine rare neurological, neurometabolic or neuromuscular diseases<\/strong><\/p>

SIMPATHIC focuses on nine rare neurological, neurometabolic, or neuromuscular diseases\u00a0<\/sup>that have few to no drug or treatment options today. Instead of developing therapies for each of 9 disease types and symptoms separately our consortium will accelerate therapy by focusing on the shared symptoms and patient needs. validated compound on two or more well-defined neuro-RD patient groups, with distinct genetic etiology but with shared clinical symptoms and molecular mechanisms.<\/p>

  • Spinocerebellar ataxia type 3;<\/li>
  • Leigh syndrome;<\/li>
  • Congenital neurotransmitter defects;<\/li>
  • Pyridoxine dependent epilepsy;<\/li>
  • Glutaric aciduria;<\/li>
  • PMM2-Congenital disorder glycosylation;<\/li>
  • Zellweger Spectrum Disorders;<\/li>
  • Myotonic dystrophy type 1;<\/li>
  • Congenital myasthenic syndrome.<\/li><\/ul>

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    Unveiling untapped potential of drug repurposing for rare diseases<\/strong><\/p>\n

    If we observe a positive effect of a drug, we will launch a clinical study in a group of patients with different disorders, but similar clinical symptoms. The novelty of this project is that we focus on several rare diseases at the time, rather than on one in particular. As existing drugs have already been tested in humans, animal studies may not be necessary, which significantly accelerates the use of drugs in new applications and reduces research costs and laboratory animal use. <\/p>

    The SIMPATHIC project aims to unveil the untapped potential of repurposing strategies for rare disorders. This involves uniting patients, researchers, and legislators, employing innovative techniques and advanced clinical trials, with the ultimate goal of delivering suitable drugs promptly to individuals with little or no available treatment options at present, just like Tim<\/span><\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t

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    SIMPATHIC Focus on several diseases Focus on nine rare neurological, neurometabolic or neuromuscular diseases SIMPATHIC focuses on nine rare neurological, neurometabolic, or neuromuscular diseases\u00a0that have few to no drug or treatment options today. Instead of developing therapies for each of 9 disease types and symptoms separately our consortium will accelerate therapy by focusing on the […]<\/p>\n","protected":false},"author":8,"featured_media":0,"parent":0,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"","meta":{"footnotes":""},"class_list":["post-386","page","type-page","status-publish","hentry"],"featured_image_src":null,"featured_image_src_square":null,"_links":{"self":[{"href":"https:\/\/simpathic.eu\/index.php?rest_route=\/wp\/v2\/pages\/386","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/simpathic.eu\/index.php?rest_route=\/wp\/v2\/pages"}],"about":[{"href":"https:\/\/simpathic.eu\/index.php?rest_route=\/wp\/v2\/types\/page"}],"author":[{"embeddable":true,"href":"https:\/\/simpathic.eu\/index.php?rest_route=\/wp\/v2\/users\/8"}],"replies":[{"embeddable":true,"href":"https:\/\/simpathic.eu\/index.php?rest_route=%2Fwp%2Fv2%2Fcomments&post=386"}],"version-history":[{"count":3,"href":"https:\/\/simpathic.eu\/index.php?rest_route=\/wp\/v2\/pages\/386\/revisions"}],"predecessor-version":[{"id":390,"href":"https:\/\/simpathic.eu\/index.php?rest_route=\/wp\/v2\/pages\/386\/revisions\/390"}],"wp:attachment":[{"href":"https:\/\/simpathic.eu\/index.php?rest_route=%2Fwp%2Fv2%2Fmedia&parent=386"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}