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Taylor &Francis OnlineÀÌ ¹ßÇ¥ÇÑ Ãֽе¥ÀÌÅÍ(2025³â)¿¡ µû¸£¸é ±ÙÀ§Ãà¼ºÃø»ö°æÈÁõ(ALS)ÀÇ Àü ¼¼°è ºÎ´ãÀº ²ÙÁØÈ÷ Áõ°¡ÇÒ °ÍÀ¸·Î ¿¹ÃøµË´Ï´Ù. 2022³â¿¡ º¸°íµÈ ¾à 3¸¸ 2,893°Ç¿¡¼ 10% ÀÌ»ó Áõ°¡ÇÏ¿© 2030³â¿¡´Â ¾à 3¸¸ 6,308°Ç¿¡ ´ÞÇÒ °ÍÀ¸·Î ¿¹ÃøµË´Ï´Ù. ÀÌ·¯ÇÑ Áõ°¡ Ãß¼¼´Â ¹ßº´·ü Áõ°¡¸¦ ¹Ý¿µÇÒ »Ó¸¸ ¾Æ´Ï¶ó, Áø´Ü ´É·ÂÀÇ Çâ»ó°ú Áúº´¿¡ ´ëÇÑ ÀνÄÀÌ ³ô¾ÆÁ³À½À» º¸¿©ÁÝ´Ï´Ù.
À̸¦ Áö¿øÇϵí, 12elveInsightÀÇ 2023³â ½ÃÀå ºÐ¼®¿¡ µû¸£¸é ¹Ì±¹, ¿µ±¹, µ¶ÀÏ, ÇÁ¶û½º, ÀÌÅ»¸®¾Æ, ½ºÆäÀÎ, ÀϺ»À» Æ÷ÇÔÇÑ ÁÖ¿ä 7°³ ½ÃÀå¿¡¼ ALSÀÇ À¯º´·üÀº ¾à 6¸¸ 7,000°ÇÀ̾ú½À´Ï´Ù. ÀÌ´Â ±âÁ¸ ÀÇ·á ½Ã½ºÅÛ ³»¿¡¼ È¿°úÀûÀÎ Ä¡·á °³ÀÔ¿¡ ´ëÇÑ ¼ö¿ä°¡ ¸¹´Ù´Â °ÍÀ» º¸¿©ÁÝ´Ï´Ù. MND È£ÁÖ(2024³â) ÀÚ·á¿¡ µû¸£¸é ¸ÅÀÏ ÃÖ¼Ò 2¸íÀÌ ¿îµ¿½Å°æÁúȯ(MND)À¸·Î Áø´Ü¹Þ°í ÀÖÀ¸¸ç, ÇöÀç ¾à 2,688¸íÀÌ ÀÌ ÁúȯÀ» ¾Î°í ÀÖ´Â °ÍÀ¸·Î ³ªÅ¸³ª, Á¢±Ù¼º ³ôÀº Ä¡·á Àü·«ÀÌ ½Ã±ÞÈ÷ ÇÊ¿äÇÑ °ÍÀ¸·Î ³ªÅ¸³µ½À´Ï´Ù.
µå¹°Áö¸¸ ½É°¢ÇÑ À¯Àü¼º ½Å°æ±ÙÀ°ÁúȯÀΠô¼ö¼º ±ÙÀ§ÃàÁõ(SMA)ÀÇ °æ¿ì, ô¼ö¼º ±ÙÀ§ÃàÁõ Àç´Ü(2023³â)Àº ÇöÀç ¹Ì±¹¿¡¼ 1¸¸-2¸¸5,000¸íÀÌ ¾Î°í ÀÖ´Â °ÍÀ¸·Î Ãß»êÇϰí ÀÖ½À´Ï´Ù. ÀÌ ÁúȯÀÇ Ãâ»ý½Ã À¯º´·üÀº 6,000-1¸¸ ¸í´ç 1¸í²Ã·Î, Á¶±â °ËÁø°ú Á¶±â °³ÀÔÀÇ Á߿伺ÀÌ °Á¶µÇ°í ÀÖ½À´Ï´Ù. À¯ÀüÀÚ Ä¡·á, ƯÈ÷ ¾Æµ¥³ëºÎ¼öü ¹ÙÀÌ·¯½º º¤Å͸¦ ÀÌ¿ëÇÑ SMN1 À¯ÀüÀÚ Ä¡È¯Àº Àå±â ¿¹Èĸ¦ ÇöÀúÈ÷ °³¼±ÇÏ°í °æ¿ì¿¡ µû¶ó¼´Â ¿îµ¿±â´ÉÀ» ȸº¹½ÃŰ´Â ȹ±âÀûÀÎ ÇØ°áÃ¥À¸·Î µîÀåÇß½À´Ï´Ù.
¿îµ¿½Å°æÁúȯ, ƯÈ÷ ALS¿Í SMAÀÇ À¯º´·ü Áõ°¡´Â Àü ¼¼°è MND Ä¡·áÁ¦ ½ÃÀåÀÇ ±Þ°ÝÇÑ ¼ºÀåÀ» °¡¼ÓÇÏ´Â ÁÖ¿ä ¿äÀÎÀ¸·Î ÀÛ¿ëÇϰí ÀÖ½À´Ï´Ù. ±Ù·Â ¾àÈ, È£Èí Àå¾Ö, ±Ã±ØÀûÀÎ ¿îµ¿ ´É·Â »ó½ÇÀ» Ư¡À¸·Î ÇÏ´Â ÀÌ ÁøÇ༺ ½Å°æÁúȯÀº Á¡Á¡ ´õ ½É°¢ÇÑ °øÁß º¸°Ç ¹®Á¦·Î Àνĵǰí ÀÖ½À´Ï´Ù. ±× °á°ú, Çõ½ÅÀûÀÎ Áúº´ º¯Çü Ä¡·á¿¡ ´ëÇÑ ¼ö¿ä°¡ Áõ°¡Çϰí ÀÖ½À´Ï´Ù.
Ä¡·á Ãø¸é¿¡¼´Â MND Ä¡·áÀÇ ÆÐ·¯´ÙÀÓ ÀüȯÀÌ ÁøÇà ÁßÀÔ´Ï´Ù. ALS´Â Áõ»ó ¿Ïȸ¦ ³Ñ¾î ½Å°æ¼¼Æ÷ ÅðÇàÀÇ ÁøÇàÀ» ´ÊÃßµµ·Ï ¼³°èµÈ ½Å°æº¸È£Á¦ °³¹ß·Î ÃÊÁ¡ÀÌ È®´ëµÇ°í ÀÖ½À´Ï´Ù. SMA¿¡¼ À¯ÀüÀÚ Ä¡·á´Â Ç¥ÁØ Ä¡·á¸¦ ÀçÁ¤ÀÇÇϰí, Ä¡·áÀÇ °¡´É¼ºÀ» Á¦°øÇϸç, ¸¹Àº ȯÀÚµéÀÇ Áúº´ ±ËÀûÀ» ¹Ù²Ù°í ÀÖ½À´Ï´Ù.
µ¿½Ã¿¡ ±ÔÁ¦ ȯ°æÀº ±â¼ú Çõ½ÅÀ» ´õ¿í ÃËÁøÇÏ´Â ¿ªÇÒÀ» Çϰí ÀÖ½À´Ï´Ù. ÃÖ±Ù ¹Ì±¹ ½ÄǰÀǾ౹(FDA)°ú À¯·´ÀǾàǰû(EMA)ÀÇ ½ÂÀÎÀº ½ÃÀå¿¡ ´ëÇÑ ½Å·Ú°¨À» ³ôÀ̰í ÀÖ½À´Ï´Ù. ƯÈ÷ Zolgensma(R)(onasemnogene abeparvovec)ÀÇ ½ÂÀÎÀº À¯ÀüÀÚ Ä¡·á Á¢±Ù¹ýÀÇ À¯È¿¼ºÀ» ÀÔÁõÇϰí, ¿îµ¿½Å°æ º¸Á¸°ú ±ÙÀ° ±â´É °È¸¦ ¸ñÇ¥·Î ÇÏ´Â Â÷¼¼´ë Ä¡·áÁ¦¿¡ ´ëÇÑ ÅõÀÚ¸¦ °¡¼ÓÈÇß½À´Ï´Ù.
½ÃÀå °³¹ßÀ» ÃßÁøÇÏ´Â ±â¾÷µµ ÷´Ü Ç¥ÀûÄ¡·áÁ¦ÀÇ ½Ç¿ëȸ¦ À§ÇØ ¿¬±¸°³¹ßÀ» °ÈÇϰí ÀÖ½À´Ï´Ù. ¿¹¸¦ µé¾î Biogen Inc.´Â 2023³â 4¿ù, SOD1(Superoxide Dismutase 1) À¯ÀüÀÚ º¯À̰¡ È®ÀÎµÈ ¼ºÀÎÀÇ ALS Ä¡·á¸¦ ÀûÀÀÁõÀ¸·Î Çϴ ô¼ö°³» ÁÖ»çÁ¦ QALSODY(TM)(ÀϹݸí ÅäÆä¸£¼¾)¿¡ ´ëÇÑ FDA ½Å¼Ó ½ÂÀÎÀ» ȹµæÇß½À´Ï´Ù. ÀÌ °áÁ¤Àº ½Å°æ¼¼Æ÷ ¼Õ»ó°ú °ü·ÃµÈ ¹ÙÀÌ¿À¸¶Ä¿ÀÎ Ç÷Àå ´º·ÎÇʶó¸àÆ® °æ¼â(NfL) ¼öÁØÀÇ °¨¼Ò¿¡ ±â¹ÝÇÑ °ÍÀ¸·Î, ¹ÙÀÌ¿À¸¶Ä¿ ±â¹Ý Ä¡·á¹ý °³¹ß¿¡¼ Áß¿äÇÑ ÁøÀüÀ» ÀÌ·é °ÍÀÔ´Ï´Ù.
°á·ÐÀûÀ¸·Î ALS¿Í SMAÀÇ ¹ßº´·ü Áõ°¡¿Í ÀÎÁöµµ Áõ°¡´Â Ä¡·á Çõ½ÅÀÇ ¹ßÀü°ú ¾çÈ£ÇÑ ±ÔÁ¦Àû ¹è°æ°ú °áÇÕÇÏ¿© °ß°íÇÏ°í ¿ªµ¿ÀûÀÎ ¿îµ¿½Å°æÁúȯ Ä¡·áÁ¦ ½ÃÀåÀ» Çü¼ºÇϰí ÀÖ½À´Ï´Ù. ÀÌ·¯ÇÑ ¹ßÀüÀ¸·Î ÀÌ ºÐ¾ß´Â 2025-2032³âÀÇ ¿¹Ãø ±â°£ Áß Áö¼ÓÀûÀÎ ¼ºÀå°ú Áö¼ÓÀûÀÎ °úÇÐÀû Çõ½ÅÀ» ±â´ëÇÒ ¼ö ÀÖ½À´Ï´Ù.
±×·¯³ª ³ôÀº Ä¡·áºñ, ±ÔÁ¦±â°ü ¹× Á¤ºÎÀÇ ¾ö°ÝÇÑ °¡À̵å¶óÀÎ µîÀÌ ¿îµ¿½Å°æÁúȯ Ä¡·áÁ¦ ½ÃÀåÀÇ ¼ºÀåÀ» Á¦ÇÑÇÏ´Â ÁÖ¿ä Á¦¾à¿äÀÎÀ¸·Î ÀÛ¿ëÇϰí ÀÖ½À´Ï´Ù.
¿îµ¿½Å°æÁúȯ Ä¡·áÁ¦ ½ÃÀåÀÇ ºÎ¹® ºÐ¼® :
¿îµ¿½Å°æÁúȯ Ä¡·áÁ¦ ½ÃÀåÀÇ ¾à¹° ºÐ·ùº°·Î´Â »ýÁ¸ ¿îµ¿½Å°æ(SMN) ÀÛ¿ëÁ¦ Ä«Å×°í¸®°¡ 2024³â °¡Àå Å« ½ÃÀå Á¡À¯À²À» Â÷ÁöÇÒ °ÍÀ¸·Î ÃßÁ¤µË´Ï´Ù. ÀÌ Ä«Å×°í¸®ÀÇ ¼ºÀåÀº ÁַΠô¼ö¼º ±ÙÀ§ÃàÁõ(SMA)ÀÇ À¯º´·ü Áõ°¡¿Í SMN1À» Ç¥ÀûÀ¸·Î ÇÏ´Â Ä¡·á¿Í °ü·ÃµÈ ¸Å·ÂÀûÀÎ Ä¡·á È¿°ú¿¡ ±âÀÎÇÕ´Ï´Ù. SMN ´Ü¹éÁúÀº ¿îµ¿ ´º·±ÀÇ »ýÁ¸°ú ±â´É¿¡ ÇʼöÀûÀÎ ¿îµ¿ ´º·±ÀÇ »ýÁ¸°ú ±â´É¿¡ ÇʼöÀûÀÔ´Ï´Ù. SMN ÀÛ¿ëÁ¦´Â SMN ´Ü¹éÁúÀÇ »ý¼ºÀ» °ÈÇÏ¿© ¿îµ¿ ±â´ÉÀ» À¯ÁöÇϰí, Áúº´ÀÇ ÁøÇàÀ» Áö¿¬½Ã۰í, SMA ȯÀÚÀÇ Àü¹ÝÀûÀÎ »îÀÇ ÁúÀ» °³¼±ÇÏ´Â µ¥ µµ¿òÀÌ µË´Ï´Ù.
¿¹¸¦ µé¾î NHS England(2023³â)¿¡ µû¸£¸é ¿µ±¹¿¡¼´Â ¸Å³â ¾à 70¸íÀÇ SMA ¾ÆÀ̰¡ ž´Ï´Ù. ¸¶Âù°¡Áö·Î È£ÁÖÀηùÀ¯ÀüÇÐȸ(2024³â)´Â 1¸¸ ¸í Áß 1¸íÀÌ SMA·Î ž°í, 40¸í Áß 1¸íÀÌ °ü·Ã À¯ÀüÀÚ º¯ÀÌÀÇ °Ç°ÇÑ º¸±ÕÀÚ¶ó°í º¸°íÇϰí ÀÖÀ¸¸ç, ÀÌ´Â ½É°¢ÇÑ À§Çè Áý´ÜÀ̸ç È¿°úÀûÀÎ °³ÀÔÀÌ ½Ã±ÞÈ÷ ÇÊ¿äÇÏ´Ù´Â °ÍÀ» °Á¶Çϰí ÀÖ½À´Ï´Ù.
SMNÀ» Ç¥ÀûÀ¸·Î ÇÏ´Â Ä¡·á¹ýÀÇ ÁÖ¸ñÇÒ ¸¸ÇÑ ÀåÁ¡ Áß Çϳª´Â SMAÀÇ ´Ù¾çÇÑ º´Çü(ÁßÁõ 1ÇüºÎÅÍ °æÁõ 3Çü±îÁö)¿¡ °ÉÃÄ ÀÓ»óÀû È¿°ú¸¦ ÃÊ·¡ÇÏ´Â º´Çü °£ È¿´ÉÀ» Á¦°øÇÒ ¼ö ÀÖ´Ù´Â Á¡ÀÔ´Ï´Ù. ¿¹¸¦ µé¾î °¡Àå ½É°¢ÇÑ 1Çü SMA Áø´ÜÀ» ¹ÞÀº ¿µ¾ÆµéÀº Ä¡·á¹ÞÁö ¾ÊÀº °æ¿ì µå¹°°Ô º¸Á¶ ¾øÀÌ ¾É´Â °Í°ú °°Àº ¹ß´Þ ´Ü°è¿¡ µµ´ÞÇÏ´Â µî ¿îµ¿ ±â´ÉÀÌ ÇöÀúÇÏ°Ô °³¼±µÈ °ÍÀ¸·Î ³ªÅ¸³µ½À´Ï´Ù.
SMN ÀÛ¿ëÁ¦ Ä¡·á´Â ¿îµ¿ ±â´É °³¼± ¿Ü¿¡µµ °æ±¸¿ë ¾àÁ¦À̱⠶§¹®¿¡ ƯÈ÷ ¼Ò¾Æ ȯÀÚ¿¡¼ Ä¡·á Á¢±Ù¼º°ú ¼øÀÀµµ¸¦ ³ôÀÏ ¼ö ÀÖ´Ù´Â ÀåÁ¡ÀÌ ÀÖ½À´Ï´Ù. À̸¦ ÅëÇØ Àå±âÀûÀÎ Ä¡·á °ü¸®°¡ º¸´Ù ½ÇÇà °¡´ÉÇϰí ȯÀÚ Ä£ÈÀûÀÌ¾î¼ Ä¡·á ¼øÀÀµµ¸¦ ³ôÀ̰í Ä¡·á °á°ú¸¦ °³¼±ÇÏ´Â µ¥ ±â¿©ÇÕ´Ï´Ù. SMN ÀÛ¿ëÁ¦´Â SMN ´Ü¹éÁú ¼öÄ¡¸¦ Áõ°¡½ÃÄÑ SMAÀÇ ÁøÇàÀ» Áö¿¬½Ã۰í, °ü·Ã ½Å°æ±Ù Áõ»óÀ» ¿ÏÈÇϸç, ƯÈ÷ Á¶±â ¹ßº´ÀÇ ÁßÁõ ȯÀÚ¿¡¼ »ýÁ¸ ±â°£À» ¿¬ÀåÇÏ´Â µ¥ µµ¿òÀÌ µË´Ï´Ù.
ÀÌ Ä«Å×°í¸®ÀÇ ¼ºÀå¿¡ ´õ¿í ¹ÚÂ÷¸¦ °¡Çϰí ÀÖ´Â °ÍÀº R&D ÅõÀÚ Áõ°¡¿Í ±ÔÁ¦ÀÇ ¹ßÀüÀÔ´Ï´Ù. ¾÷°è ÁÖ¿ä ¾÷üµéÀº È¿°ú¿Í ÆíÀǼºÀ» Çâ»ó½Ã۱â À§ÇØ Â÷¼¼´ë SMN Ç¥Àû Ä¡·áÁ¦¸¦ Àû±ØÀûÀ¸·Î °³¹ßÇϰí ÀÖ½À´Ï´Ù. ±× ´ëÇ¥ÀûÀÎ ¿¹°¡ 2025³â 2¿ù Roche°¡ ¹Ì±¹ ½ÄǰÀǾ౹(FDA)À¸·ÎºÎÅÍ Evrysdi(R)(¸®½ºÁöÇÁ¶÷) Á¤Á¦ Á¦Á¦ÀÇ ½Å¾à½ÂÀνÅû(NDA)À» ½ÂÀÎ¹Þ¾Ò´Ù°í ¹ßÇ¥ÇÑ °ÍÀÔ´Ï´Ù. Evrysdi´Â SMN2 pre-mRNA splicing modifier·Î, SMN ´Ü¹éÁúÀÇ °á¼ÕÀ» ÃÊ·¡ÇÏ´Â ¿°»öü 5q À¯ÀüÀÚÀÇ µ¹¿¬º¯ÀÌ·Î ÀÎÇØ ¹ß»ýÇÏ´Â SMA¸¦ Ä¡·áÇϱâ À§ÇØ °í¾ÈµÇ¾ú½À´Ï´Ù. ÀÌ Á¤Á¦´Â Áý¿¡¼ º¹¿ëÇÒ ¼ö ÀÖ´Â °æ±¸¿ë Àü½Å Ä¡·áÁ¦·Î, º´¿ø¿¡¼ ¼ö¾×À» ¸ÂÀ» Çʿ䰡 ¾ø¾î ȯÀÚ¿Í °¡Á·¿¡°Ô º¸´Ù Ä£±ÙÇÑ Ä¡·áÁ¦°¡ µÉ ¼ö ÀÖ½À´Ï´Ù.
ÀÌ¿Í °°ÀÌ À§¿Í °°Àº ¿äÀεéÀÌ ½ÃÀå ºÎ¹®À» ²ø¾î¿Ã¸®°í, ³ª¾Æ°¡ Àü ¼¼°è ¿îµ¿½Å°æÁúȯ Ä¡·áÁ¦ ½ÃÀå Àüü¸¦ È®´ëÇÒ °ÍÀ¸·Î º¸ÀÔ´Ï´Ù.
ºÏ¹Ì´Â ÁÖ¿ä ±¸Á¶Àû ¹× Àü·«Àû ¿ìÀ§ÀÇ Á¶ÇÕÀ¸·Î ÀÎÇØ 2024³â ¼¼°è ¿îµ¿½Å°æÁúȯ Ä¡·áÁ¦ ½ÃÀå¿¡¼ °¡Àå Å« Á¡À¯À²À» Â÷ÁöÇÒ °ÍÀ¸·Î ¿¹ÃøµË´Ï´Ù. ÀÌ Áö¿ªÀÇ ¿ìÀ§´Â ÁַΠô¼ö¼º ±ÙÀ§ÃàÁõ(SMA), ±ÙÀ§Ãà¼ºÃø»ö°æÈÁõ(ALS)°ú °°Àº ¿îµ¿½Å°æÁúȯÀÇ À¯º´·ü Áõ°¡·Î ÀÎÇØ È¿°úÀûÀÎ Ä¡·á ¼Ö·ç¼Ç¿¡ ´ëÇÑ ÇコÄÉ¾î ½Ã½ºÅÛ¿¡ ´ëÇÑ ¼ö¿ä°¡ Áö¼ÓÀûÀ¸·Î Áõ°¡Çϰí Àֱ⠶§¹®ÀÔ´Ï´Ù. ¶ÇÇÑ ÀÌ Áö¿ªÀº ÁÖ¿ä »ý¸í°øÇÐ ¹× Á¦¾à ±â¾÷ÀÌ Çõ½ÅÀûÀÎ Ä¡·á¹ý °³¹ß, ÀÓ»ó½ÃÇè ¹× »ó¿ëȸ¦ À§ÇØ Àû±ØÀûÀ¸·Î ³ë·ÂÇϰí ÀÖÀ¸¸ç, źźÇÑ Á¦¾à ȯ°æÀÇ ÇýÅÃÀ» ´©¸®°í ÀÖ½À´Ï´Ù. ½ÅÁ¦Ç° ½ÂÀÎ ¼Óµµ´Â ¿©ÀüÈ÷ ³ôÀ¸¸ç, ´õ¿í À¯¸®ÇÑ ±ÔÁ¦ ÇÁ·¹ÀÓ¿öÅ©¿¡ ÀÇÇØ Áö¿øµÇ°í ÀÖ½À´Ï´Ù. ƯÈ÷ ¹Ì±¹ ½ÄǰÀǾ౹(FDA)°ú °°Àº ±â°üÀº Èñ±ÍÁúȯ Ä¡·áÁ¦¿¡ ´ëÇÑ ½Å¼Ó ½ÂÀÎ °æ·Î¸¦ µµÀÔÇÏ¿© MND¸¦ À§ÇÑ ÃÖ÷´Ü Ä¡·áÁ¦ÀÇ ½Å¼ÓÇÑ °ø±ÞÀ» Áö¿øÇϰí ÀÖ½À´Ï´Ù. Áúº´ ºÎ´ã Áõ°¡, °·ÂÇÑ R&D Ȱµ¿, Àû±ØÀûÀÎ ±ÔÁ¦ ´ç±¹ÀÇ Áö¿ø µî ÀÌ·¯ÇÑ ¿äÀεéÀ» Á¾ÇÕÇÏ¸é ºÏ¹Ì´Â ¿îµ¿½Å°æÁúȯ Ä¡·áÁ¦ ½ÃÀåÀÇ ¼¼°è ¸®´õ·Î¼ ÀÔÁö¸¦ ±»È÷°í ÀÖ½À´Ï´Ù.
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Motor Neuron Disease Treatment Market by Drug Class (NMDA (N-methyl-D-aspartate) Receptor Antagonist, SOD1 Gene Mutation Agonist, Alpha-2 Adrenergic Agonist, Survival Motor Neuron (SMN) Agonist, and Others), Route of Administration (Oral and Parenteral), Indication (Amyotrophic Lateral Sclerosis (ALS), Spinal Muscular Atrophy (SMA), and Others), Distribution Channel (Hospitals and Retail Pharmacies and Online Pharmacies), and Geography (North America, Europe, Asia-Pacific, and Rest of the World) is expected to grow at a steady CAGR forecast till 2032 owing to the growing prevalence of motor neuron diseases and increasing investments and innovations in the research and development of MND drugs.
The motor neuron disease treatment market is estimated to grow at a CAGR of 5.87% during the forecast period from 2025 to 2032. The demand for motor neuron disease (MND) treatment is experiencing significant growth, driven by the rising prevalence of motor neuron disorders such as Amyotrophic Lateral Sclerosis (ALS), Spinal Muscular Atrophy (SMA), and others. Increasing awareness about the importance of early and accurate diagnosis is further accelerating market momentum. Moreover, a notable shift toward biological therapies which offer targeted and more effective treatment options is transforming the therapeutic landscape. This, combined with increasing investments and continuous innovation in the research and development of MND therapies, is fostering a highly favorable environment for market expansion. These factors collectively position the motor neuron disease treatment market for robust and sustained growth throughout the forecast period from 2025 to 2032.
Motor Neuron Disease Treatment Market Dynamics:
According to recent data published by Taylor & Francis Online (2025), the global burden of Amyotrophic Lateral Sclerosis (ALS) is projected to grow steadily. From approximately 32,893 reported cases in 2022, the number was expected to increase by over 10%, reaching an estimated 36,308 cases by 2030. This upward trend not only reflected a rise in incidence rates but also highlighted improvements in diagnostic capabilities and heightened disease awareness.
Supporting this, a 2023 market analysis by DelveInsight revealed that across the seven major markets including the United States, United Kingdom, Germany, France, Italy, Spain, and Japan, the prevalence of ALS stood at around 67,000 cases. This indicated a substantial demand for effective therapeutic interventions within well-established healthcare systems. Regionally, Australia mirrors this growing burden, data from MND Australia (2024), showed that at least two individuals were diagnosed with motor neuron disease (MND) each day, with approximately 2,688 people who were currently living with that condition, underscoring the pressing need for accessible treatment strategies.
In the case of Spinal Muscular Atrophy (SMA), a rare but severe genetic neuromuscular disorder, the Spinal Muscular Atrophy Foundation (2023) estimated that between 10,000 and 25,000 individuals were currently affected in the United States. The condition had a birth prevalence of approximately 1 in every 6,000 to 10,000 live births, emphasizing the critical importance of early screening and intervention. Gene therapy, particularly SMN1 gene replacement using adeno-associated viral vectors, has emerged as a transformative solution, significantly improving long-term outcomes and, in some instances, restoring motor function.
Collectively, the rising prevalence of motor neuron diseases particularly ALS and SMA is a key driver behind the rapid expansion of the global MND treatment market. These progressive neurological disorders, characterized by muscle weakness, respiratory compromise, and eventual loss of mobility, are increasingly recognized as serious public health concerns. As a result, there is mounting demand for innovative, disease-modifying therapies.
Therapeutically, the MND treatment landscape is undergoing a paradigm shift. In ALS, the focus is expanding beyond symptomatic relief to the development of neuroprotective agents designed to slow the progression of neuronal degeneration. In SMA, gene therapy has redefined the standard of care, offering potentially curative benefits and altering the disease trajectory for many patients.
At the same time, the regulatory environment is becoming more supportive of innovation. Recent approvals by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have bolstered confidence in the market. Notably, the approval of Zolgensma(R) (onasemnogene abeparvovec) has validated gene therapy approaches and accelerated investment in next-generation treatments that target motor neuron preservation and muscle function enhancement.
Market players are also intensifying their research and development efforts to commercialize advanced targeted therapies. For example, in April 2023, Biogen Inc. received FDA accelerated approval for QALSODY(TM) (tofersen), an intrathecal injection indicated for the treatment of ALS in adults with a confirmed mutation in the superoxide dismutase 1 (SOD1) gene. The decision was based on reductions in plasma neurofilament light chain (NfL) levels, a biomarker linked to neuronal damage, marking a significant step forward in biomarker-driven therapeutic development.
In conclusion, the growing incidence and improved recognition of ALS and SMA, coupled with advancements in therapeutic innovation and a favorable regulatory backdrop, are collectively shaping a robust and dynamic motor neuron disease treatment market. These developments position the sector for sustained growth and ongoing scientific breakthroughs throughout the forecast period from 2025 to 2032.
However, the high cost of the treatment and stringent guidelines by regulatory bodies and governments, among others are some of the key constraints that may limit the growth of the motor neuron disease treatment market.
Motor Neuron Disease Treatment Market Segment Analysis:
Motor Neuron Disease Treatment Market by Drug Class (NMDA (N-methyl-D-aspartate) Receptor Antagonist, SOD1 Gene Mutation Agonist, Alpha-2 Adrenergic Agonist, Survival Motor Neuron (SMN) Agonist, and Others), Route of Administration (Oral and Parenteral), Indication (Amyotrophic Lateral Sclerosis (ALS), Spinal Muscular Atrophy (SMA), and Others), Distribution Channel (Hospital and Retail Pharmacies and Online Pharmacies), and Geography (North America, Europe, Asia-Pacific, and Rest of the World)
In the drug class segment of the motor neuron disease treatment market, the survival motor neuron (SMN) agonist category is estimated to account for the largest market share in 2024. The growth of this category is primarily driven by the increasing prevalence of Spinal Muscular Atrophy (SMA) and the compelling therapeutic benefits associated with SMN1-targeted treatments. The SMN protein is critical for the survival and function of motor neurons, which are essential for voluntary muscle movement. SMN agonists work by enhancing the production of SMN protein, helping to preserve motor function, delay disease progression, and improve the overall quality of life for patients living with SMA.
For instance, according to NHS England (2023), approximately 70 children were born with SMA each year in the United Kingdom. Similarly, the Human Genetics Society of Australia (2024) reported that 1 in 10,000 individuals were born with SMA, while 1 in 40 people were healthy carriers of the associated genetic mutation highlighting the significant at-risk population and the urgent need for effective interventions.
One notable advantage of SMN-targeted therapies is their ability to provide cross-type efficacy, offering clinical benefits across different forms of SMA, from severe Type 1 to milder Type 3. For example, infants diagnosed with Type 1 SMA typically the most severe form have demonstrated remarkable improvements in motor function, including reaching developmental milestones such as sitting unassisted, an outcome that is rare in untreated cases.
In addition to motor function improvements, SMN agonist therapies offer the benefit of oral formulations, enhancing treatment accessibility and compliance, especially among pediatric patients. This makes long-term treatment management more feasible and patient-friendly, contributing to better adherence and improved outcomes. By boosting SMN protein levels, SMN agonists help slow the progression of SMA, mitigate its associated neuromuscular symptoms, and prolong survival, particularly in early-onset and severe cases.
Further fueling this category's growth is the increase in R&D investments and regulatory advancements. Key industry players are actively developing next-generation SMN-targeted treatments to improve efficacy and convenience. A notable example is from February 2025, when Roche announced that the U.S. Food and Drug Administration (FDA) approved a New Drug Application (NDA) for a tablet formulation of Evrysdi(R) (risdiplam). Evrysdi is an SMN2 pre-mRNA splicing modifier, designed to treat SMA caused by mutations in the chromosome 5q gene, which result in a deficiency of the SMN protein. This tablet formulation offers an oral, systemic treatment alternative that can be taken at home, reducing the need for hospital-based infusions and making care more accessible for patients and families.
Thus, the factors mentioned above are likely to boost the market segment and thereby increase the overall market of motor neuron disease treatment across the globe.
North America is expected to dominate the overall motor neuron disease treatment market:
North America is expected to hold the largest share of the global motor neuron disease treatment market in 2024, driven by a combination of key structural and strategic advantages. This regional dominance is primarily attributed to the rising prevalence of motor neuron disorders such as Spinal Muscular Atrophy (SMA) and Amyotrophic Lateral Sclerosis (ALS), which continue to place increasing demand on healthcare systems for effective treatment solutions. Additionally, the region benefits from a robust pharmaceutical landscape, with leading biotech and pharmaceutical companies actively engaged in the development, clinical trials, and commercialization of innovative therapies. The pace of new product approvals remains high, further supported by favorable regulatory frameworks. Notably, agencies such as the U.S. Food and Drug Administration (FDA) have implemented accelerated approval pathways for rare disease drugs, helping to fast-track the availability of cutting-edge treatments for MNDs. Collectively, these factors such as the rising disease burden, strong R&D activity, and proactive regulatory support are solidifying North America's position as the global leader in the motor neuron disease treatment market.
According to recent data from the Centers for Disease Control and Prevention (CDC, 2025), approximately 33,000 cases of Amyotrophic Lateral Sclerosis (ALS) were recorded in the U.S. in 2022, with the number projected to rise by over 10% to exceed 36,000 cases by 2030. Similarly, the American Medical Association (2024) reported that 1 in every 14,694 newborns in the U.S. was diagnosed with Spinal Muscular Atrophy (SMA) in 2024, underscoring the growing burden of motor neuron diseases (MNDs) in North America.
To combat these progressive and often fatal disorders, motor neuron-targeted therapies are increasingly being adopted, particularly those focused on replacing defective or missing SMN1 genes, thereby providing functional copies to preserve motor neuron health and slow muscular degeneration. Supporting this trend, the National Institutes of Health (2022), noted that in Canada, between 1 in 6,000 to 10,000 babies were born with SMA annually, highlighting a regional need for timely diagnosis and advanced interventions.
The rising incidence of SMA is a key driver of growth in the North American motor neuron disease treatment market, as it has led to increased demand for disease-modifying therapies. Given that SMA often manifests in infancy or early childhood, there is a heightened urgency for early genetic screening and innovative treatments such as SMN-targeted gene therapy, which has spurred substantial R&D investment and therapeutic development.
A significant enabler of market expansion in the region is the proactive regulatory environment. The U.S. Food and Drug Administration (FDA) has accelerated progress in the MND treatment landscape by offering fast-track and breakthrough therapy designations for promising drugs. Notably, Zolgensma(R), a gene therapy for SMA, received accelerated approval, marking a breakthrough in disease-modifying treatment and further validating gene-based approaches.
In addition to regulatory support, ongoing innovation by leading U.S.-based pharmaceutical companies is reinforcing market momentum. For example, in April 2023, Biogen Inc. announced that the FDA had granted accelerated approval for QALSODY(TM) (tofersen), an intrathecal injection indicated for ALS patients with a confirmed SOD1 gene mutation. The approval was based on the drug's ability to reduce plasma neurofilament light chain (NfL) levels, a biomarker linked to neuronal damage, making QALSODY available in the U.S. market as a new targeted treatment option.
Collectively, the combination of increasing disease prevalence, continuous product innovation, regulatory support, and strategic initiatives by major industry players is expected to drive robust growth in the North American motor neuron disease treatment market throughout the forecast period from 2025 to 2032.
Motor Neuron Disease Treatment Market Key Players:
Some of the key market players operating in the motor neuron disease treatment market include Biogen, Mitsubishi Chemical Group Corporation, F. Hoffmann-La Roche Ltd., Otsuka Holdings Co., Ltd., Sanofi, Ionis Pharmaceuticals, Inc., Novartis AG, EDW Pharma, Inc., PTC Therapeutics, Inc., Chugai Pharmaceutical Co., Ltd., AMYLYX Pharmaceuticals, and others.
Recent Developmental Activities in the Motor Neuron Disease Treatment Market:
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