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  1. sleepreviewmag.com › sleep-treatmentsPitolisant Improves Fatigue in Myotonic Dystrophy Patients

    1 day ago · Summary: Harmony Biosciences presented data from a phase 2 signal detection study indicating that pitolisant reduced excessive daytime sleepiness (EDS) and fatigue in adults with myotonic dystrophy type 1 (DM1). The study showed greater improvements in EDS and fatigue scores for patients receiving pitolisant compared to placebo, particularly at ...

  2. www.nature.com › articles › s43587/024/00645-9Cell type mapping of inflammatory muscle diseases ... - Nature

    3 days ago · When including all cell type profiles based on snRNA-seq and marker gene expression, we could identify MNs from type 1 fibers (ATP2A2, referred to here as type 1 MNs) and type 2 fibers (ATP2A1 ...

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  4. physoc.onlinelibrary.wiley.com › doi › 10BREATHE DMD: boosting respiratory efficacy after therapeutic ...

    3 days ago · A recent meta-analysis has demonstrated that exercise has no significant impact on extensor muscle strength but does improve muscle endurance during walking in patients with facio-scapulo-humeral dystrophy and myotonic dystrophy (Gianola et al., 2020).

  5. www.gimjournal.org › article › S1098-3600(24)00078-9Myotonic dystrophy type 1 testing, 2024 revision: A technical ...

    2 days ago · Myotonic dystrophy type 1 (DM1) is a form of muscular dystrophy causing progressive muscle loss and weakness. Although clinical features can manifest at any age, it is the most common form of muscular dystrophy with onset in adulthood. DM1 is an autosomal dominant condition, resulting from an unstable CTG expansion in the 3′-untranslated region of the myotonic dystrophy protein kinase (DMPK ...

  6. finance.yahoo.com › news › harmony-biosciencesHARMONY BIOSCIENCES PRESENTS POSITIVE DATA FOR PITOLISANT IN ...

    2 days ago · Myotonic dystrophy Type 1 (DM1) is the most common form of adult-onset muscular dystrophy. It is a genetic disorder inherited in an autosomal-dominant pattern. Latest estimates suggest a ...

  7. www.mun-h-center.se › duchenne-muscular-dystrophyDuchenne Muscular Dystrophy - Public_munhcenter

    3 days ago · Duchenne Muscular Dystrophy This is the text from the oral care programme for people with Duchenne muscular dystrophy (DMD) Mun-H-Center, a Swedish orofacial resource center for rare diseases and a specialist dental clinic, has gathered knowledge and experience over many years regarding the clinical care of patients with Duchenne Muscular Dystrophy (DMD).

  8. www.myotonic.org › mdf-regional-conference-seattle-waMDF Regional Conference, Seattle, WA | Myotonic Dystrophy ...

    6 days ago · Westin Seattle - Starting at $239+ tax/night. Booking Deadline: May 10th. 1900 Fifth Avenue, Seattle, WA 98101. The conference will be held at this location. To book by phone, please call +1 206-728-1000. Click here to complete your booking online.

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