{"id":6789,"date":"2025-06-25T10:43:11","date_gmt":"2025-06-25T10:43:11","guid":{"rendered":"https:\/\/cureclcn4.org\/wissenschaftliche-konferenz-cure-clcn4-highlights-fuer-familien\/"},"modified":"2025-06-25T10:43:14","modified_gmt":"2025-06-25T10:43:14","slug":"wissenschaftliche-konferenz-cure-clcn4-highlights-fuer-familien","status":"publish","type":"post","link":"https:\/\/cureclcn4.org\/de\/wissenschaftliche-konferenz-cure-clcn4-highlights-fuer-familien\/","title":{"rendered":"Wissenschaftliche Konferenz Cure CLCN4 &#8211; Highlights f\u00fcr Familien"},"content":{"rendered":"<p>[et_pb_section fb_built=&#8220;1&#8243; admin_label=&#8220;Get Involved&#8220; _builder_version=&#8220;4.24.2&#8243; background_color=&#8220;#FFFFFF&#8220; background_image=&#8220;https:\/\/cureclcn4.org\/wp-content\/uploads\/2020\/08\/charity-13.png&#8220; parallax=&#8220;on&#8220; width=&#8220;100%&#8220; custom_padding=&#8220;0|0px|0px|0px|false|false&#8220; use_custom_width=&#8220;on&#8220; custom_width_px=&#8220;1200px&#8220; 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text_font_size=&#8220;5px&#8220; text_line_height=&#8220;2em&#8220; header_font=&#8220;Faustina|600|||||||&#8220; header_text_color=&#8220;#0e7162&#8243; header_font_size=&#8220;57px&#8220; header_line_height=&#8220;1.3em&#8220; width=&#8220;94.4%&#8220; min_height=&#8220;83px&#8220; custom_margin=&#8220;-19px|||19px|false|false&#8220; header_font_size_tablet=&#8220;50px&#8220; header_font_size_phone=&#8220;40px&#8220; header_font_size_last_edited=&#8220;on|phone&#8220; global_colors_info=&#8220;{}&#8220;]<\/p>\n<h1 style=\"text-align: center;\"><\/h1>\n<h1 style=\"text-align: center;\">Wissenschaftliche Konferenz Cure CLCN4 &#8211; Highlights f\u00fcr Familien<\/h1>\n<p>[\/et_pb_text][et_pb_image src=&#8220;https:\/\/cureclcn4.org\/wp-content\/uploads\/2025\/06\/04258F6F-F016-42AE-9A6D-75C73A4A5351_1_105_c-copy.jpeg&#8220; title_text=&#8220;04258F6F-F016-42AE-9A6D-75C73A4A5351_1_105_c copy&#8220; _builder_version=&#8220;4.27.4&#8243; _module_preset=&#8220;default&#8220; global_colors_info=&#8220;{}&#8220;][\/et_pb_image][et_pb_text _builder_version=&#8220;4.27.4&#8243; text_font=&#8220;||||||||&#8220; text_font_size=&#8220;20px&#8220; text_line_height=&#8220;2em&#8220; header_font=&#8220;Faustina||||||||&#8220; header_text_color=&#8220;#0e7162&#8243; header_font_size=&#8220;60px&#8220; header_line_height=&#8220;1.3em&#8220; text_orientation=&#8220;justified&#8220; min_height=&#8220;52px&#8220; custom_margin=&#8220;|5px||5px|false|true&#8220; custom_padding=&#8220;|10px||10px|false|true&#8220; global_colors_info=&#8220;{}&#8220;]<\/p>\n<p style=\"font-weight: 400;\"><span>Wir haben k\u00fcrzlich f\u00fchrende Wissenschaftler und Kliniker zusammengebracht, um die neuesten Forschungsergebnisse und Ideen zum besseren Verst\u00e4ndnis der <em>CLCN4-Krankheit<\/em> auszutauschen. <\/span><span>Hier sind einige wichtige Erkenntnisse der Konferenz in einfacher Sprache:<\/span><\/p>\n<p style=\"text-align: center;\">[\/et_pb_text][et_pb_divider color=&#8220;#12876f&#8220; divider_weight=&#8220;5px&#8220; _builder_version=&#8220;4.25.0&#8243; _module_preset=&#8220;default&#8220; min_height=&#8220;6px&#8220; global_colors_info=&#8220;{}&#8220;][\/et_pb_divider][\/et_pb_column][\/et_pb_row][et_pb_row _builder_version=&#8220;4.27.4&#8243; background_color=&#8220;#ffffff&#8220; width=&#8220;100%&#8220; max_width=&#8220;1090px&#8220; min_height=&#8220;41px&#8220; custom_margin=&#8220;10px||10px||false|false&#8220; custom_padding=&#8220;0px||0px||false|false&#8220; global_colors_info=&#8220;{}&#8220;][et_pb_column type=&#8220;4_4&#8243; admin_label=&#8220;S\u00e4ule&#8220; _builder_version=&#8220;4.27.4&#8243; custom_padding=&#8220;0px||0px||false|false&#8220; custom_padding_tablet=&#8220;0px||&#8220; custom_padding_phone=&#8220;&#8220; custom_padding_last_edited=&#8220;on|desktop&#8220; global_colors_info=&#8220;{}&#8220; padding_tablet=&#8220;0px||&#8220; padding_last_edited=&#8220;on|tablet&#8220; custom_padding__hover=&#8220;|||&#8220; padding_phone=&#8220;0px||&#8220;][et_pb_text _builder_version=&#8220;4.27.4&#8243; text_font=&#8220;||||||||&#8220; text_font_size=&#8220;20px&#8220; text_line_height=&#8220;2em&#8220; header_font=&#8220;Faustina|600|||||||&#8220; header_text_color=&#8220;#0e7162&#8243; header_font_size=&#8220;60px&#8220; header_line_height=&#8220;1.3em&#8220; header_2_font=&#8220;|700|||||||&#8220; header_2_text_color=&#8220;#0e7162&#8243; header_2_font_size=&#8220;30px&#8220; text_orientation=&#8220;center&#8220; module_alignment=&#8220;center&#8220; min_height=&#8220;41px&#8220; custom_margin=&#8220;||0px|0px|false|false&#8220; custom_padding=&#8220;|25px|0px|25px|false|false&#8220; header_font_size_tablet=&#8220;50px&#8220; header_font_size_phone=&#8220;40px&#8220; header_font_size_last_edited=&#8220;on|phone&#8220; global_colors_info=&#8220;{}&#8220;]<\/p>\n<h2 style=\"text-align: left;\">Was wir \u00fcber das <em>CLCN4-Gen<\/em> erfahren<\/h2>\n<p>[\/et_pb_text][\/et_pb_column][\/et_pb_row][et_pb_row column_structure=&#8220;1_2,1_2&#8243; _builder_version=&#8220;4.27.4&#8243; _module_preset=&#8220;default&#8220; background_color=&#8220;#FFFFFF&#8220; width=&#8220;100%&#8220; min_height=&#8220;376px&#8220; custom_margin=&#8220;0px||0px||false|false&#8220; custom_padding=&#8220;0px|0px|0px||false|false&#8220; global_colors_info=&#8220;{}&#8220;][et_pb_column type=&#8220;1_2&#8243; _builder_version=&#8220;4.27.4&#8243; _module_preset=&#8220;default&#8220; custom_padding=&#8220;0px||0px||false|false&#8220; global_colors_info=&#8220;{}&#8220;][et_pb_text _builder_version=&#8220;4.27.4&#8243; text_font=&#8220;||||||||&#8220; text_font_size=&#8220;16px&#8220; text_line_height=&#8220;2em&#8220; header_font=&#8220;Faustina||||||||&#8220; header_text_color=&#8220;#0e7162&#8243; header_font_size=&#8220;60px&#8220; header_line_height=&#8220;1.3em&#8220; text_orientation=&#8220;justified&#8220; min_height=&#8220;328px&#8220; custom_margin=&#8220;10px|5px|0px|5px|false|true&#8220; custom_padding=&#8220;0px|10px|0px|10px|false|false&#8220; global_colors_info=&#8220;{}&#8220;]<\/p>\n<ul>\n<li>Die <em>CLCN4-Krankheit<\/em> wird durch Varianten oder &#8218;Ver\u00e4nderungen&#8216; im <em>CLCN4-Gen<\/em> verursacht. Das <em>CLCN4-Gen<\/em> gibt Anweisungen zur Herstellung eines Proteins namens ClC-4. Es wird angenommen, dass dieses Protein eine wichtige Rolle bei der <strong>Funktion des Gehirns und der Nervenzellen<\/strong> spielt <strong>.<\/strong>  <\/li>\n<li><strong>Ver\u00e4nderungen im <em>CLCN4-Gen<\/em> wirken sich darauf aus, wie das ClC-4-Protein<\/strong> in den Zellen des K\u00f6rpers einer betroffenen Person <strong>funktioniert<\/strong>.<\/li>\n<li>Die Forschung best\u00e4tigt, dass <strong>ClC-4 eng mit einem &#8222;Partnerprotein&#8220; namens ClC-3 zusammenarbeitet<\/strong>, und wenn eines davon nicht richtig funktioniert, kann das andere versuchen, dies zu kompensieren.<\/li>\n<li>Vor kurzem wurden auch <strong>neue Partnerproteine<\/strong> (TMEM9A und TMEM9B genannt) entdeckt. Diese k\u00f6nnten dazu beitragen, die Funktionsweise von ClC-4 im Gehirn zu kontrollieren und k\u00f6nnten neue Ziele f\u00fcr zuk\u00fcnftige Behandlungen sein. <\/li>\n<\/ul>\n<p>[\/et_pb_text][\/et_pb_column][et_pb_column type=&#8220;1_2&#8243; _builder_version=&#8220;4.27.4&#8243; _module_preset=&#8220;default&#8220; custom_padding=&#8220;0px||0px||false|false&#8220; global_colors_info=&#8220;{}&#8220;][et_pb_image src=&#8220;https:\/\/cureclcn4.org\/wp-content\/uploads\/2025\/06\/1.png&#8220; title_text=&#8220;DNA&#8220; align=&#8220;center&#8220; _builder_version=&#8220;4.27.4&#8243; _module_preset=&#8220;default&#8220; max_width=&#8220;75%&#8220; custom_margin=&#8220;0px||||false|false&#8220; global_colors_info=&#8220;{}&#8220;][\/et_pb_image][\/et_pb_column][\/et_pb_row][et_pb_row _builder_version=&#8220;4.27.4&#8243; background_color=&#8220;#ffffff&#8220; width=&#8220;100%&#8220; max_width=&#8220;1100px&#8220; min_height=&#8220;70px&#8220; height=&#8220;100px&#8220; custom_margin=&#8220;10px||5px||false|false&#8220; custom_padding=&#8220;0px||0px||false|false&#8220; global_colors_info=&#8220;{}&#8220;][et_pb_column type=&#8220;4_4&#8243; _builder_version=&#8220;4.27.4&#8243; custom_padding=&#8220;0px||0px||false|false&#8220; custom_padding_tablet=&#8220;0px||&#8220; custom_padding_phone=&#8220;&#8220; custom_padding_last_edited=&#8220;on|desktop&#8220; global_colors_info=&#8220;{}&#8220; padding_tablet=&#8220;0px||&#8220; padding_last_edited=&#8220;on|tablet&#8220; custom_padding__hover=&#8220;|||&#8220; padding_phone=&#8220;0px||&#8220;][et_pb_text _builder_version=&#8220;4.27.4&#8243; text_font=&#8220;||||||||&#8220; text_font_size=&#8220;20px&#8220; text_line_height=&#8220;2em&#8220; header_font=&#8220;Faustina|600|||||||&#8220; header_text_color=&#8220;#0e7162&#8243; header_font_size=&#8220;60px&#8220; header_line_height=&#8220;1.3em&#8220; header_2_font=&#8220;|700|||||||&#8220; header_2_text_color=&#8220;#0e7162&#8243; header_2_font_size=&#8220;30px&#8220; text_orientation=&#8220;center&#8220; width=&#8220;92.2%&#8220; min_height=&#8220;87px&#8220; height=&#8220;100px&#8220; max_height=&#8220;1000px&#8220; custom_margin=&#8220;0px|19px|0px|19px|false|false&#8220; custom_padding=&#8220;0px||0px||false|false&#8220; header_font_size_tablet=&#8220;50px&#8220; header_font_size_phone=&#8220;40px&#8220; header_font_size_last_edited=&#8220;on|phone&#8220; global_colors_info=&#8220;{}&#8220;]<\/p>\n<h2 style=\"text-align: left;\">Tiermodelle k\u00f6nnen uns helfen, <em>CLCN4-bedingte<\/em> Erkrankungen besser zu verstehen<\/h2>\n<p>[\/et_pb_text][\/et_pb_column][\/et_pb_row][et_pb_row column_structure=&#8220;1_2,1_2&#8243; _builder_version=&#8220;4.27.4&#8243; _module_preset=&#8220;default&#8220; background_color=&#8220;#FFFFFF&#8220; width=&#8220;100%&#8220; min_height=&#8220;346px&#8220; custom_margin=&#8220;0px||0px||false|false&#8220; custom_padding=&#8220;0px|0px|0px||false|false&#8220; global_colors_info=&#8220;{}&#8220;][et_pb_column type=&#8220;1_2&#8243; _builder_version=&#8220;4.27.4&#8243; _module_preset=&#8220;default&#8220; custom_padding=&#8220;0px||0px||false|false&#8220; global_colors_info=&#8220;{}&#8220;][et_pb_text _builder_version=&#8220;4.27.4&#8243; text_font=&#8220;||||||||&#8220; text_font_size=&#8220;16px&#8220; text_line_height=&#8220;2em&#8220; header_font=&#8220;Faustina||||||||&#8220; header_text_color=&#8220;#0e7162&#8243; header_font_size=&#8220;60px&#8220; header_line_height=&#8220;1.3em&#8220; text_orientation=&#8220;justified&#8220; min_height=&#8220;343px&#8220; custom_margin=&#8220;5px|5px|0px|5px|false|true&#8220; custom_padding=&#8220;0px|10px|0px|10px|false|false&#8220; global_colors_info=&#8220;{}&#8220;]<\/p>\n<ul style=\"font-weight: 400;\">\n<li><span>Die k\u00fcrzlich entwickelten <strong><em>Clcn4-Rattenmodelle<\/em> <\/strong>zeigen Symptome, die denen einiger Patienten \u00e4hneln, wie <strong>Hyperaktivit\u00e4t, Aufmerksamkeitsst\u00f6rungen und Koordinationsprobleme<\/strong>. Erste Ergebnisse deuten auch auf Lern- und Ged\u00e4chtnisprobleme hin, aber es muss noch mehr getan werden, um dies zu best\u00e4tigen.   <\/span><\/li>\n<li><span>Wissenschaftler versuchen auch, ein <strong><em>CLCN4-Zebrafischmodell <\/em><\/strong> zu entwickeln, um <em>CLCN4-bedingte<\/em>Erkrankungen besser zu verstehen. Zebrab\u00e4rblinge sind transparent, entwickeln sich schnell und sind billiger zu halten als Nagetiere. Das k\u00f6nnte den Forschern helfen, Hunderte von Medikamenten zu testen, um diejenigen zu finden, die helfen k\u00f6nnten. Diese Tiere helfen bereits bei der Suche nach Behandlungen f\u00fcr \u00e4hnliche Erkrankungen wie das Batten- und das Dravet-Syndrom.  <\/span><\/li>\n<\/ul>\n<p>[\/et_pb_text][\/et_pb_column][et_pb_column type=&#8220;1_2&#8243; _builder_version=&#8220;4.27.4&#8243; _module_preset=&#8220;default&#8220; custom_padding=&#8220;0px||0px||false|false&#8220; global_colors_info=&#8220;{}&#8220;][et_pb_image src=&#8220;https:\/\/cureclcn4.org\/wp-content\/uploads\/2025\/06\/2.png&#8220; _builder_version=&#8220;4.27.4&#8243; _module_preset=&#8220;default&#8220; title_text=&#8220;Tiermodell&#8220; max_width=&#8220;75%&#8220; align=&#8220;center&#8220; hover_enabled=&#8220;0&#8243; sticky_enabled=&#8220;0&#8243;][\/et_pb_image][\/et_pb_column][\/et_pb_row][et_pb_row _builder_version=&#8220;4.27.4&#8243; background_color=&#8220;#ffffff&#8220; width=&#8220;100%&#8220; max_width=&#8220;1100px&#8220; min_height=&#8220;38px&#8220; custom_margin=&#8220;10px|auto|10px|auto|false|false&#8220; custom_padding=&#8220;0px||0px||false|false&#8220; global_colors_info=&#8220;{}&#8220;][et_pb_column type=&#8220;4_4&#8243; _builder_version=&#8220;4.27.4&#8243; custom_padding=&#8220;0px||0px||false|false&#8220; custom_padding_tablet=&#8220;0px||&#8220; custom_padding_phone=&#8220;&#8220; custom_padding_last_edited=&#8220;on|desktop&#8220; global_colors_info=&#8220;{}&#8220; padding_tablet=&#8220;0px||&#8220; padding_last_edited=&#8220;on|tablet&#8220; custom_padding__hover=&#8220;|||&#8220; padding_phone=&#8220;0px||&#8220;][et_pb_text _builder_version=&#8220;4.27.4&#8243; text_font=&#8220;||||||||&#8220; text_font_size=&#8220;20px&#8220; text_line_height=&#8220;2em&#8220; header_font=&#8220;Faustina|600|||||||&#8220; header_text_color=&#8220;#0e7162&#8243; header_font_size=&#8220;60px&#8220; header_line_height=&#8220;1.3em&#8220; header_2_font=&#8220;|700|||||||&#8220; header_2_text_color=&#8220;#0e7162&#8243; header_2_font_size=&#8220;30px&#8220; text_orientation=&#8220;center&#8220; width=&#8220;92.2%&#8220; min_height=&#8220;40px&#8220; custom_margin=&#8220;0px|10px|0px|10px|false|false&#8220; custom_padding=&#8220;0px||0px||false|false&#8220; header_font_size_tablet=&#8220;50px&#8220; header_font_size_phone=&#8220;40px&#8220; header_font_size_last_edited=&#8220;on|phone&#8220; global_colors_info=&#8220;{}&#8220;]<\/p>\n<h2 style=\"text-align: left;\">Untersuchung von <em>CLCN4-Varianten<\/em> <\/h2>\n<p>[\/et_pb_text][\/et_pb_column][\/et_pb_row][et_pb_row column_structure=&#8220;1_2,1_2&#8243; _builder_version=&#8220;4.27.4&#8243; _module_preset=&#8220;default&#8220; background_color=&#8220;#FFFFFF&#8220; width=&#8220;100%&#8220; min_height=&#8220;202px&#8220; custom_margin=&#8220;0px||0px||false|false&#8220; custom_padding=&#8220;0px||0px||false|false&#8220; global_colors_info=&#8220;{}&#8220;][et_pb_column type=&#8220;1_2&#8243; _builder_version=&#8220;4.27.4&#8243; _module_preset=&#8220;default&#8220; custom_padding=&#8220;0px||0px||false|false&#8220; global_colors_info=&#8220;{}&#8220;][et_pb_text _builder_version=&#8220;4.27.4&#8243; text_font=&#8220;||||||||&#8220; text_font_size=&#8220;16px&#8220; text_line_height=&#8220;2em&#8220; header_font=&#8220;Faustina||||||||&#8220; header_text_color=&#8220;#0e7162&#8243; header_font_size=&#8220;60px&#8220; header_line_height=&#8220;1.3em&#8220; text_orientation=&#8220;justified&#8220; custom_margin=&#8220;0px|5px|0px|5px|false|true&#8220; custom_padding=&#8220;0px|10px|0px|10px|false|true&#8220; global_colors_info=&#8220;{}&#8220;]<\/p>\n<ul>\n<li>Nicht alle genetischen Ver\u00e4nderungen (Varianten) im <em>CLCN4-Gen<\/em> f\u00fchren zu einer <em>CLCN4-bedingten<\/em> Erkrankung. Es gibt viele gesunde, nicht betroffene Personen mit Varianten im <em>CLCN4-Gen<\/em>, deren ClC-4-Protein einwandfrei funktioniert. <\/li>\n<li>Es wird daran gearbeitet, <strong>zu verstehen, welche Varianten am wahrscheinlichsten Symptome verursachen<\/strong> und wie genau sie die Gehirnfunktion beeinflussen k\u00f6nnen.<\/li>\n<li>Diese Arbeit ist wichtig, um <strong>neue Varianten zu klassifizieren<\/strong>, <strong>die Diagnose zu unterst\u00fctzen<\/strong> und <strong>Behandlungsentscheidungen<\/strong> in der Zukunft zu treffen.<\/li>\n<\/ul>\n<p>[\/et_pb_text][\/et_pb_column][et_pb_column type=&#8220;1_2&#8243; _builder_version=&#8220;4.27.4&#8243; _module_preset=&#8220;default&#8220; custom_padding=&#8220;0px||0px||false|false&#8220; global_colors_info=&#8220;{}&#8220;][et_pb_image src=&#8220;https:\/\/cureclcn4.org\/wp-content\/uploads\/2025\/06\/3.png&#8220; title_text=&#8220;Forscher&#8220; align=&#8220;center&#8220; _builder_version=&#8220;4.27.4&#8243; _module_preset=&#8220;default&#8220; max_width=&#8220;75%&#8220; global_colors_info=&#8220;{}&#8220;][\/et_pb_image][\/et_pb_column][\/et_pb_row][et_pb_row _builder_version=&#8220;4.27.4&#8243; background_color=&#8220;#ffffff&#8220; width=&#8220;100%&#8220; max_width=&#8220;1100px&#8220; min_height=&#8220;31px&#8220; custom_margin=&#8220;10px||10px||false|false&#8220; custom_padding=&#8220;0px||0px||false|false&#8220; global_colors_info=&#8220;{}&#8220;][et_pb_column type=&#8220;4_4&#8243; _builder_version=&#8220;4.27.4&#8243; custom_padding=&#8220;0px||0px||false|false&#8220; custom_padding_tablet=&#8220;0px||&#8220; custom_padding_phone=&#8220;&#8220; custom_padding_last_edited=&#8220;on|desktop&#8220; global_colors_info=&#8220;{}&#8220; padding_tablet=&#8220;0px||&#8220; padding_last_edited=&#8220;on|tablet&#8220; custom_padding__hover=&#8220;|||&#8220; padding_phone=&#8220;0px||&#8220;][et_pb_text _builder_version=&#8220;4.27.4&#8243; text_font=&#8220;||||||||&#8220; text_font_size=&#8220;20px&#8220; text_line_height=&#8220;2em&#8220; header_font=&#8220;Faustina|600|||||||&#8220; header_text_color=&#8220;#0e7162&#8243; header_font_size=&#8220;60px&#8220; header_line_height=&#8220;1.3em&#8220; header_2_font=&#8220;|700|||||||&#8220; header_2_text_color=&#8220;#0e7162&#8243; header_2_font_size=&#8220;30px&#8220; text_orientation=&#8220;center&#8220; width=&#8220;92.2%&#8220; min_height=&#8220;26px&#8220; custom_margin=&#8220;0px|19px|0px|19px|false|false&#8220; custom_padding=&#8220;0px||0px||false|false&#8220; header_font_size_tablet=&#8220;50px&#8220; header_font_size_phone=&#8220;40px&#8220; header_font_size_last_edited=&#8220;on|phone&#8220; global_colors_info=&#8220;{}&#8220;]<\/p>\n<h2 style=\"text-align: left;\">Die menschliche Seite: mehr als eine Diagnose<\/h2>\n<p>[\/et_pb_text][\/et_pb_column][\/et_pb_row][et_pb_row column_structure=&#8220;1_2,1_2&#8243; _builder_version=&#8220;4.27.4&#8243; _module_preset=&#8220;default&#8220; background_color=&#8220;#FFFFFF&#8220; width=&#8220;100%&#8220; min_height=&#8220;259px&#8220; custom_margin=&#8220;10px|auto|10px|auto|false|false&#8220; custom_padding=&#8220;0px|0px|10px||false|false&#8220; global_colors_info=&#8220;{}&#8220;][et_pb_column type=&#8220;1_2&#8243; _builder_version=&#8220;4.27.4&#8243; _module_preset=&#8220;default&#8220; custom_padding=&#8220;0px||0px||false|false&#8220; global_colors_info=&#8220;{}&#8220;][et_pb_text _builder_version=&#8220;4.27.4&#8243; text_font=&#8220;||||||||&#8220; text_font_size=&#8220;16px&#8220; text_line_height=&#8220;2em&#8220; header_font=&#8220;Faustina||||||||&#8220; header_text_color=&#8220;#0e7162&#8243; header_font_size=&#8220;60px&#8220; header_line_height=&#8220;1.3em&#8220; text_orientation=&#8220;justified&#8220; min_height=&#8220;239px&#8220; custom_margin=&#8220;10px|5px|0px|5px|false|true&#8220; custom_padding=&#8220;0px|10px|0px|10px|false|false&#8220; global_colors_info=&#8220;{}&#8220;]<\/p>\n<ul>\n<li>Das <em>CLCN4-Syndrom<\/em> ist nicht nur eine Krankheit, sondern eine Gruppe von <em>verwandten<\/em> Erfahrungen. Es handelt sich um eine neurologische Entwicklungsst\u00f6rung mit einer <strong>breiten Palette von Symptomen<\/strong>, darunter Lernschwierigkeiten, Kommunikationsprobleme, Epilepsie und Bewegungs- oder Verhaltensst\u00f6rungen. <\/li>\n<li>Der <strong>Schweregrad und die spezifischen Merkmale k\u00f6nnen erheblich variieren<\/strong>, selbst bei Personen aus derselben Familie.<\/li>\n<li>Was alle Menschen mit einer <em>CLCN4-bedingten<\/em> Erkrankung gemeinsam haben, ist die <strong>au\u00dfergew\u00f6hnliche Belastbarkeit<\/strong>, das <strong>Einf\u00fchlungsverm\u00f6gen<\/strong> und die F\u00e4higkeit, den Menschen in ihrer Umgebung <strong>Liebe und Verbundenheit<\/strong> entgegenzubringen.<\/li>\n<\/ul>\n<p>[\/et_pb_text][\/et_pb_column][et_pb_column type=&#8220;1_2&#8243; _builder_version=&#8220;4.27.4&#8243; _module_preset=&#8220;default&#8220; custom_padding=&#8220;0px||0px||false|false&#8220; global_colors_info=&#8220;{}&#8220;][et_pb_image src=&#8220;https:\/\/cureclcn4.org\/wp-content\/uploads\/2025\/06\/4.png&#8220; title_text=&#8220;Gl\u00fcckliches Kind&#8220; _builder_version=&#8220;4.27.4&#8243; _module_preset=&#8220;default&#8220; max_width=&#8220;75%&#8220; custom_margin=&#8220;0px|0px|0px|0px|false|false&#8220; hover_enabled=&#8220;0&#8243; global_colors_info=&#8220;{}&#8220; align=&#8220;center&#8220; sticky_enabled=&#8220;0&#8243; module_alignment=&#8220;center&#8220;][\/et_pb_image][\/et_pb_column][\/et_pb_row][et_pb_row _builder_version=&#8220;4.27.4&#8243; background_color=&#8220;#ffffff&#8220; width=&#8220;100%&#8220; max_width=&#8220;1100px&#8220; min_height=&#8220;46px&#8220; custom_margin=&#8220;10px||10px||false|false&#8220; custom_padding=&#8220;0px||0px||false|false&#8220; global_colors_info=&#8220;{}&#8220;][et_pb_column type=&#8220;4_4&#8243; _builder_version=&#8220;4.27.4&#8243; custom_padding=&#8220;0px||0px||false|false&#8220; custom_padding_tablet=&#8220;0px||&#8220; custom_padding_phone=&#8220;&#8220; custom_padding_last_edited=&#8220;on|desktop&#8220; global_colors_info=&#8220;{}&#8220; padding_tablet=&#8220;0px||&#8220; padding_last_edited=&#8220;on|tablet&#8220; custom_padding__hover=&#8220;|||&#8220; padding_phone=&#8220;0px||&#8220;][et_pb_text _builder_version=&#8220;4.27.4&#8243; text_font=&#8220;||||||||&#8220; text_font_size=&#8220;20px&#8220; text_line_height=&#8220;2em&#8220; header_font=&#8220;Faustina|600|||||||&#8220; header_text_color=&#8220;#0e7162&#8243; header_font_size=&#8220;60px&#8220; header_line_height=&#8220;1.3em&#8220; header_2_font=&#8220;|700|||||||&#8220; header_2_text_color=&#8220;#0e7162&#8243; header_2_font_size=&#8220;30px&#8220; text_orientation=&#8220;center&#8220; width=&#8220;92.2%&#8220; min_height=&#8220;26px&#8220; custom_margin=&#8220;0px|19px|0px|19px|false|false&#8220; custom_padding=&#8220;0px||0px||false|false&#8220; header_font_size_tablet=&#8220;50px&#8220; header_font_size_phone=&#8220;40px&#8220; header_font_size_last_edited=&#8220;on|phone&#8220; global_colors_info=&#8220;{}&#8220;]<\/p>\n<h2 style=\"text-align: left;\">Das Potenzial von Gentherapien<\/h2>\n<p>[\/et_pb_text][\/et_pb_column][\/et_pb_row][et_pb_row column_structure=&#8220;1_2,1_2&#8243; _builder_version=&#8220;4.27.4&#8243; _module_preset=&#8220;default&#8220; background_color=&#8220;#FFFFFF&#8220; width=&#8220;100%&#8220; min_height=&#8220;282px&#8220; custom_margin=&#8220;0px|auto|0px|auto|false|false&#8220; custom_padding=&#8220;0px|0px|0px||false|false&#8220; global_colors_info=&#8220;{}&#8220;][et_pb_column type=&#8220;1_2&#8243; _builder_version=&#8220;4.27.4&#8243; _module_preset=&#8220;default&#8220; custom_padding=&#8220;0px||0px||false|false&#8220; global_colors_info=&#8220;{}&#8220;][et_pb_text _builder_version=&#8220;4.27.4&#8243; text_font=&#8220;||||||||&#8220; text_font_size=&#8220;16px&#8220; text_line_height=&#8220;2em&#8220; header_font=&#8220;Faustina||||||||&#8220; header_text_color=&#8220;#0e7162&#8243; header_font_size=&#8220;60px&#8220; header_line_height=&#8220;1.3em&#8220; text_orientation=&#8220;justified&#8220; min_height=&#8220;224px&#8220; custom_margin=&#8220;0px|5px|0px|5px|false|true&#8220; custom_padding=&#8220;|10px||10px|false|false&#8220; global_colors_info=&#8220;{}&#8220;]<\/p>\n<ul>\n<li>Ein wichtiger Schwerpunkt der Konferenz war das <strong>Potenzial von Gentherapien<\/strong>, also Behandlungen, die auf bestimmte Fehler in den Genen einer Person abzielen.<\/li>\n<li>Neue Hilfsmittel k\u00f6nnen nun individuelle genetische Ver\u00e4nderungen analysieren und feststellen, welche davon auf eine Therapie ansprechen k\u00f6nnten, wie z.B. Antisense-Oligonukleotide (ASOs), die bereits Menschen mit anderen seltenen Krankheiten helfen.<\/li>\n<li>Es wird auch mit <strong>von Patienten stammenden Stammzellen (iPSCs<\/strong> ) gearbeitet, um Nervenzellen im Labor zu erzeugen und Behandlungen direkt an ihnen zu testen.<\/li>\n<li>Diese Therapien f\u00fcr <em>CLCN4-bedingte<\/em> Erkrankungen befinden sich noch in einem fr\u00fchen Stadium, aber die Technologie entwickelt sich schnell, und mehrere Teams arbeiten jetzt daran, zu verstehen, ob und wie diese Therapien bei <em>CLCN4-bedingten<\/em> Erkrankungen angewendet werden k\u00f6nnten.<\/li>\n<\/ul>\n<p>[\/et_pb_text][\/et_pb_column][et_pb_column type=&#8220;1_2&#8243; _builder_version=&#8220;4.27.4&#8243; _module_preset=&#8220;default&#8220; custom_padding=&#8220;0px||0px||false|false&#8220; global_colors_info=&#8220;{}&#8220;][et_pb_image src=&#8220;https:\/\/cureclcn4.org\/wp-content\/uploads\/2025\/06\/5.png&#8220; title_text=&#8220;Gentherapie&#8220; align=&#8220;center&#8220; _builder_version=&#8220;4.27.4&#8243; _module_preset=&#8220;default&#8220; max_width=&#8220;75%&#8220; custom_margin=&#8220;10px||||false|false&#8220; global_colors_info=&#8220;{}&#8220;][\/et_pb_image][\/et_pb_column][\/et_pb_row][et_pb_row _builder_version=&#8220;4.27.4&#8243; background_color=&#8220;#ffffff&#8220; width=&#8220;100%&#8220; max_width=&#8220;1100px&#8220; min_height=&#8220;42px&#8220; custom_margin=&#8220;10px||10px||false|false&#8220; custom_padding=&#8220;0px||0px||false|false&#8220; global_colors_info=&#8220;{}&#8220;][et_pb_column type=&#8220;4_4&#8243; _builder_version=&#8220;4.27.4&#8243; custom_padding=&#8220;0px||0px||false|false&#8220; custom_padding_tablet=&#8220;0px||&#8220; custom_padding_phone=&#8220;&#8220; custom_padding_last_edited=&#8220;on|desktop&#8220; global_colors_info=&#8220;{}&#8220; padding_tablet=&#8220;0px||&#8220; padding_last_edited=&#8220;on|tablet&#8220; custom_padding__hover=&#8220;|||&#8220; padding_phone=&#8220;0px||&#8220;][et_pb_text _builder_version=&#8220;4.27.4&#8243; text_font=&#8220;||||||||&#8220; text_font_size=&#8220;20px&#8220; text_line_height=&#8220;2em&#8220; header_font=&#8220;Faustina|600|||||||&#8220; header_text_color=&#8220;#0e7162&#8243; header_font_size=&#8220;60px&#8220; header_line_height=&#8220;1.3em&#8220; header_2_font=&#8220;|700|||||||&#8220; header_2_text_color=&#8220;#0e7162&#8243; header_2_font_size=&#8220;30px&#8220; text_orientation=&#8220;center&#8220; width=&#8220;92.2%&#8220; min_height=&#8220;26px&#8220; custom_margin=&#8220;0px|19px|0px|19px|false|false&#8220; custom_padding=&#8220;0px||||false|false&#8220; header_font_size_tablet=&#8220;50px&#8220; header_font_size_phone=&#8220;40px&#8220; header_font_size_last_edited=&#8220;on|phone&#8220; global_colors_info=&#8220;{}&#8220;]<\/p>\n<h2 style=\"text-align: left;\">Bessere klinische Versorgung und Unterst\u00fctzung<\/h2>\n<p>[\/et_pb_text][\/et_pb_column][\/et_pb_row][et_pb_row column_structure=&#8220;1_2,1_2&#8243; _builder_version=&#8220;4.27.4&#8243; _module_preset=&#8220;default&#8220; background_color=&#8220;#FFFFFF&#8220; width=&#8220;100%&#8220; min_height=&#8220;240px&#8220; custom_margin=&#8220;0px|auto|0px|auto|false|false&#8220; custom_padding=&#8220;|0px|5px||false|false&#8220; global_colors_info=&#8220;{}&#8220;][et_pb_column type=&#8220;1_2&#8243; _builder_version=&#8220;4.24.2&#8243; _module_preset=&#8220;default&#8220; global_colors_info=&#8220;{}&#8220;][et_pb_text _builder_version=&#8220;4.27.4&#8243; text_font=&#8220;||||||||&#8220; text_font_size=&#8220;16px&#8220; text_line_height=&#8220;2em&#8220; header_font=&#8220;Faustina||||||||&#8220; header_text_color=&#8220;#0e7162&#8243; header_font_size=&#8220;60px&#8220; header_line_height=&#8220;1.3em&#8220; text_orientation=&#8220;justified&#8220; min_height=&#8220;214px&#8220; custom_margin=&#8220;|5px|17px|5px|false|true&#8220; custom_padding=&#8220;|10px||10px|false|false&#8220; global_colors_info=&#8220;{}&#8220;]<\/p>\n<ul>\n<li>In klinischen Vortr\u00e4gen wurde gezeigt, wie andere seltene Krankheiten von <strong>spezialisierten Kliniken<\/strong>, koordinierter Versorgung und internationalen Gesundheitsnetzwerken profitieren.<\/li>\n<li>Cure CLCN4 untersucht nun, was n\u00f6tig w\u00e4re, um eine <strong><em>CLCN4-spezifische<\/em> Klinik oder einen Pflegepfad <\/strong> einzurichten <strong>. <\/strong> <\/li>\n<li>Ein wichtiger Schritt dabei ist die <strong>Zusammenarbeit<\/strong> zwischen \u00c4rzten, Forschern und Familien.<\/li>\n<li>Unser Ziel ist es, sicherzustellen, dass Familien Zugang zu <strong>fachkundiger Betreuung<\/strong>, Informationen und <strong>Beratung<\/strong> sowie <strong>koordinierter Unterst\u00fctzung<\/strong> haben, nicht nur zu genetischen Ergebnissen.<\/li>\n<\/ul>\n<p>[\/et_pb_text][\/et_pb_column][et_pb_column type=&#8220;1_2&#8243; 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global_colors_info=&#8220;{}&#8220; padding_tablet=&#8220;0px||&#8220; padding_last_edited=&#8220;on|tablet&#8220; custom_padding__hover=&#8220;|||&#8220; padding_phone=&#8220;0px||&#8220;][et_pb_text _builder_version=&#8220;4.27.4&#8243; text_font=&#8220;||||||||&#8220; text_font_size=&#8220;20px&#8220; text_line_height=&#8220;2em&#8220; header_font=&#8220;Faustina|600|||||||&#8220; header_text_color=&#8220;#0e7162&#8243; header_font_size=&#8220;60px&#8220; header_line_height=&#8220;1.3em&#8220; header_2_font=&#8220;|700|||||||&#8220; header_2_text_color=&#8220;#0e7162&#8243; header_2_font_size=&#8220;30px&#8220; text_orientation=&#8220;center&#8220; width=&#8220;92.2%&#8220; min_height=&#8220;26px&#8220; custom_margin=&#8220;0px|19px|0px|19px|false|false&#8220; custom_padding=&#8220;0px||||false|false&#8220; header_font_size_tablet=&#8220;50px&#8220; header_font_size_phone=&#8220;40px&#8220; header_font_size_last_edited=&#8220;on|phone&#8220; global_colors_info=&#8220;{}&#8220;]<\/p>\n<h2 style=\"text-align: left;\">Was kommt als N\u00e4chstes?<\/h2>\n<p>[\/et_pb_text][\/et_pb_column][\/et_pb_row][et_pb_row column_structure=&#8220;1_2,1_2&#8243; _builder_version=&#8220;4.27.4&#8243; _module_preset=&#8220;default&#8220; background_color=&#8220;#FFFFFF&#8220; width=&#8220;100%&#8220; min_height=&#8220;291px&#8220; custom_margin=&#8220;0px|auto|25px|auto|false|false&#8220; custom_padding=&#8220;0px|0px|0px||false|false&#8220; global_colors_info=&#8220;{}&#8220;][et_pb_column type=&#8220;1_2&#8243; _builder_version=&#8220;4.24.2&#8243; _module_preset=&#8220;default&#8220; global_colors_info=&#8220;{}&#8220;][et_pb_text _builder_version=&#8220;4.27.4&#8243; text_font=&#8220;||||||||&#8220; text_font_size=&#8220;16px&#8220; text_line_height=&#8220;2em&#8220; header_font=&#8220;Faustina||||||||&#8220; header_text_color=&#8220;#0e7162&#8243; header_font_size=&#8220;60px&#8220; header_line_height=&#8220;1.3em&#8220; text_orientation=&#8220;justified&#8220; min_height=&#8220;239px&#8220; custom_margin=&#8220;0px|5px|0px|5px|false|true&#8220; custom_padding=&#8220;|10px||10px|false|false&#8220; global_colors_info=&#8220;{}&#8220;]<\/p>\n<ul>\n<li>Die Forscher planen, weitere Tests an den <em>Clcn4-Rattenmodellen<\/em> durchzuf\u00fchren, andere <em>CLCN4-bezogene<\/em> Krankheitsmodelle zu schaffen, einschlie\u00dflich Tier- und Zellmodelle, und genbasierte Therapien zu erforschen.<\/li>\n<li>Cure CLCN4 arbeitet daran, sich mit europ\u00e4ischen und britischen Netzwerken zu verbinden, um die Pflege und die Dienstleistungen zu verbessern.<\/li>\n<li>Wir werden weiterhin den Zugang zu Forschungsinstrumenten f\u00f6rdern, die Zusammenarbeit zwischen Wissenschaftlern, \u00c4rzten und Familien unterst\u00fctzen und das von Simons Searchlight betriebene <a href=\"https:\/\/cureclcn4.org\/de\/patienten-register\/\">Patientenregister<\/a> ausbauen, das eine wichtige Rolle bei der F\u00f6rderung der Forschung spielt.<\/li>\n<\/ul>\n<p>[\/et_pb_text][\/et_pb_column][et_pb_column type=&#8220;1_2&#8243; 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Zu den Themen geh\u00f6rten neue Erkenntnisse \u00fcber die Biologie der CLCN4-bedingten Erkrankung, Fortschritte bei Clcn4-Tiermodellen, das Potenzial f\u00fcr genetische Therapien und Schritte zu einer besseren klinischen Versorgung. 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