Papillon-Lefevre disease
MONDO:0009490Papillon-Lefevre syndrome (PLS) is a rare ectodermal dysplasia characterized by palmoplantar keratoderma associated with early-onset periodontitis.
Also known as: PLS, Papillon Lefèvre Syndrome, keratosis palmoplantar-periodontopathy syndrome, Keratoris palmoplantaris with periodontopathia, PALS, PAPILLON-Lefevre syndrome, Papillon-LEFèvre syndrome, Pls
26 clinical trials for this condition and its sub-types, 5 tagged with Papillon-Lefevre disease itself.
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New hope for ALS patients: experimental drug CNM-Au8 now available through expanded access
Disease control Expanded access (paused)This program provides early access to an experimental drug called CNM-Au8 for people with amyotrophic lateral sclerosis (ALS), a progressive nerve disease. Participants must be 18 or older and have a confirmed ALS diagnosis. The goal is to offer treatment to those who cannot join…
Sponsor: Clene Nanomedicine • Aim: Disease control
Last updated Jun 27, 2026 12:04 UTC
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Desperate patient gets experimental stem cells for rare nerve disease
Disease control Expanded access (ended)This study provides an experimental stem cell treatment (HB-adMSCs) to an 83-year-old man with Primary Lateral Sclerosis, a rare nerve disease that causes progressive muscle weakness. The patient's own banked stem cells are used, aiming to slow the disease. This is a single-patie…
Sponsor: Hope Biosciences Research Foundation • Aim: Disease control
Last updated Jun 27, 2026 07:52 UTC