{"id":3270,"date":"2022-01-28T14:25:00","date_gmt":"2022-01-28T14:25:00","guid":{"rendered":"https:\/\/fusfoundation.org\/?post_type=sw_disease_condition&p=3270"},"modified":"2025-06-05T11:19:14","modified_gmt":"2025-06-05T15:19:14","slug":"niemann-pick-disease","status":"publish","type":"sw_disease_condition","link":"https:\/\/www.fusfoundation.org\/diseases-and-conditions\/niemann-pick-disease\/","title":{"rendered":"Niemann-Pick Disease"},"content":{"rendered":"\n

Focused Ultrasound Therapy<\/h2>\n\n\n\n

Focused ultrasound is a noninvasive, therapeutic technology with the potential to improve the quality of life and decrease the cost of care for patients with Niemann-Pick Disease. This novel technology focuses beams of ultrasound energy precisely and accurately on targets deep in the body without damaging surrounding normal tissue.<\/p>\n\n\n\n

How it Works
<\/strong>Patients with Niemann-Pick disease lack certain enzymes for the metabolism of fat. Where the focused ultrasound beams converge, transient opening of the blood-brain barrier (BBB) can allow enhanced penetration into the central nervous system of a synthetic replacement enzyme.<\/p>\n\n\n\n

Another area being explored is using focused ultrasound for transient BBB opening to allow modified RNA penetration into the central nervous system, enabling production of the needed enzyme. Preclinical research for both of these mechanisms is being evaluated.<\/p>\n\n\n\n

Advantages
<\/strong>The primary options for treatment of Niemann-Pick Disease are primarily supportive therapies as there is no cure.

For certain patients, focused ultrasound could provide a therapeutic option for these patients. While significant work has been accomplished, there is still much to be done before this technology will be widely available.<\/p>\n\n\n\n

Focused ultrasound is noninvasive, so it does not carry added concerns like surgical wound healing or infection. It can also reach the desired target without damaging surrounding tissue and is repeatable, if necessary.<\/p>\n\n\n\n

Clinical Trials<\/h2>\n\n\n\n

At the present time, there are no clinical trials recruiting patients for focused ultrasound treatment of Niemann-Pick disease.<\/p>\n\n\n\n

The Foundation updates these pages regularly, but with the increasing number of clinical trials, we want to be sure that our audience has the latest information available. Therefore, we also added the website search information for the above trials. If you click here<\/strong><\/a>, it will take you to the latest information available from https:\/\/www.clinicaltrials.gov\/<\/a><\/strong>.\u00a0<\/p>\n\n\n\n

Regulatory Approval and Reimbursement <\/h2>\n\n\n\n

Focused ultrasound treatment for Niemann-Pick disease is not yet approved by regulatory bodies or covered by medical insurance companies.<\/p>\n\n\n\n

Notable Papers<\/h2>\n\n\n\n

Carradori D, Chen H, Werner B, Shah AS, Leonardi C, Usuelli M, Mezzenga R, Platt F, Leroux JC.\u00a0Investigating the Mechanism of Cyclodextrins in the Treatment of Niemann-Pick Disease Type C Using Crosslinked 2-Hydroxypropyl-beta-cyclodextrin<\/strong><\/a>. Small. 2020 Nov;16(46):e2004735. doi: 10.1002\/smll.202004735. Epub 2020 Oct 20. PMID: 33079457<\/p>\n\n\n\n

Click here<\/a>\u00a0for additional references from PubMed.<\/strong><\/p>\n","protected":false},"template":"","meta":{"_acf_changed":false,"_searchwp_excluded":"","_editorskit_title_hidden":false,"_editorskit_reading_time":0,"_editorskit_is_block_options_detached":false,"_editorskit_block_options_position":"{}","episode_type":"","audio_file":"","podmotor_file_id":"","podmotor_episode_id":"","cover_image":"","cover_image_id":"","duration":"","filesize":"","filesize_raw":"","date_recorded":"","explicit":"","block":"","itunes_episode_number":"","itunes_title":"","itunes_season_number":"","itunes_episode_type":"","_vp_format_video_url":"","_vp_image_focal_point":[],"footnotes":""},"sw_disease_condition_cat":[29],"sw_disease_condition_stage":[14],"class_list":["post-3270","sw_disease_condition","type-sw_disease_condition","status-publish","hentry","sw_disease_condition_cat-miscellaneous"],"acf":[],"yoast_head":"\nNiemann-Pick Disease - Focused Ultrasound Foundation<\/title>\n<meta name=\"description\" content=\"Learn how focused ultrasound, a noninvasive therapeutic technology, is being investigated to address Niemann-Pick Disease\" \/>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/www.fusfoundation.org\/diseases-and-conditions\/niemann-pick-disease\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Niemann-Pick Disease - 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