Cannabis use in myotonic dystrophy patients in Germany and USA: a pilot survey

Montagnese, Federica; White, Molly; Klein, Anke; Ståhl, Kristina; Wenninger, Stephan; Schöser, Benedikt

doi:10.1007/s00415-018-9159-2

Cited by 9 publications

(5 citation statements)

References 8 publications

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“…Acupuncture has been of some value in humans. Use of cannabinoids in people with myotonia has been associated with some positive results 48,49 . Too few dogs were treated with any single modality to draw conclusions about efficacy.…”

Section: Discussionmentioning

confidence: 99%

Clinical features of muscle stiffness in 37 dogs with concurrent naturally occurring hypercortisolism

Golinelli

Fracassi

Bianchi

et al. 2023

Veterinary Internal Medicne

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Background Severe muscle stiffness (SMS) in dogs with hypercortisolism (HC) is uncommon. Objectives To evaluate signalment, presentation, treatments, and long‐term outcomes of dogs with concurrent HC and SMS. Animals Thirty‐seven dogs. Methods Medical records of dogs with HC and concurrent SMS were recruited from 10 institutions. Clinical information, test results, therapeutic responses, and survival times were reviewed. Results All 37 dogs with HC and SMS had pituitary‐dependent hypercortisolism (PDH); 36/37 weighed <20 kg. Signs and test results were typical of PDH aside from SMS, initially diagnosed in all 4 limbs in 9, pelvic limbs of 22, and thoracic limbs of 6 dogs. Hypercortisolism and SMS were diagnosed together in 3 dogs; HC 1‐36 months before SMS in 23; SMS 1‐12 months before HC in 11. Mitotane or trilostane, given to control HC in 36/37 dogs, improved or resolved HC signs in 28; SMS did not resolve, remaining static or worsening in 31/36 dogs, mildly improving in 5/19 dogs given additional therapies. Progression of SMS included additional limbs in 10 dogs and the masticatory muscles of 2. The median survival time from diagnosis of SMS was 965 days (range, 8‐1188). Conclusions and Clinical Importance Concurrent SMS and HC is uncommon, possibly affecting only dogs with PDH. Development of SMS might occur before or after diagnosis of HC. Apart from SMS, the clinical picture and survival time of these dogs seem indistinguishable from those of dogs with HC in general. However, while muscle weakness usually resolves with HC treatment SMS does not.

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Section: Discussionmentioning

confidence: 99%

Clinical features of muscle stiffness in 37 dogs with concurrent naturally occurring hypercortisolism

Golinelli

Fracassi

Bianchi

et al. 2023

Veterinary Internal Medicne

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“…Similar to myotonia, attempts to treat myalgia and other muscular complaints led to the evaluation of cannabinoids ( cannabidiol and tetrahydrocannabinol or CBD/THC, respectively) in DM1, driven by the presence of cannabinoid receptors in muscles [29] and by modulation of both central and peripheral pain pathways [30] , [31] . In this regard, Nexien Biopharma recently filed a patent covering methods and compositions for treating patients with myotonic dystrophy (DM1 and DM2) with oral formulations of CBD and THC [32] .…”

Section: Drug Development Programsmentioning

confidence: 99%

“…Pilot survey in DM1, DM2, and congenital myotonia, with two patients per disease. Sponsor: Nexien Biopharma ( https://nexienbiopharma.com/news ) [30] , [31] , [32] Pitolisant; Wakix (small molecule/repurposing) IND-enabling phase: Phase II by 2021 Orally available to and able to cross the blood–brain barrier in animal models. Sponsor: Harmony Biosciences (www.harmonybiosciences.com/science) [57] ISIS 486178 (oligonucleotide-based therapy) Lead optimization Systemic treatment resulting in myotonia and cardiac conduction improvement, correction of splicing defects, reduction in RNA foci, and redistribution of MBNL1.…”

Section: Drug Development Programsmentioning

confidence: 99%

Myotonic dystrophy type 1 drug development: A pipeline toward the market

Pascual-Gilabert¹,

López-Castel

Artero

2021

Drug Discovery Today

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Highlights Myotonic dystrophy, a neuromuscular disease, affects at least around half a million people worldwide. Close to two dozen preclinical and clinical drug development programs active. Drugs encompass new chemical entities, repurposing, oligonucleotide, and gene therapy. Tideglusib, mexiletine, and metformin are close to reaching marketing authorization.

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“…Considering the available in vivo evidence supporting the use of cannabinoids for myopathies, apart from the phase II studies mentioned for patients with DM, the scientific background of their possible benefit in humans is restricted only to a few reports/case series 90,91,92 . That said, it is paramount to state that, in the present moment, there is no sufficient evidence to recommend a systematic cannabinoid prescription for myopathic patients.…”

Section: Cannabinoids In Muscular Diseasesmentioning

confidence: 99%

Cannabinoids in Neurology - Position paper from Scientific Departments from Brazilian Academy of Neurology

Brucki

Adoni

Almeida

et al. 2021

Arq. Neuro-Psiquiatr.

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Cannabinoids comprehend endocannabinoids, phytocannabinoids, and synthetic cannabinoids, with actions both in the central and peripherical nervous systems. A considerable amount of publications have been made in recent years, although cannabis has been known for over a thousand years. Scientific Departments from the Brazilian Academy of Neurology described evidence for medical use in their areas. Literature is constantly changing, and possible new evidence can emerge in the next days or months. Prescription of these substances must be discussed with patients and their families, with knowledge about adverse events and their efficacy.

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Cannabis use in myotonic dystrophy patients in Germany and USA: a pilot survey

Cited by 9 publications

References 8 publications

Clinical features of muscle stiffness in 37 dogs with concurrent naturally occurring hypercortisolism

Clinical features of muscle stiffness in 37 dogs with concurrent naturally occurring hypercortisolism

Myotonic dystrophy type 1 drug development: A pipeline toward the market

Cannabinoids in Neurology - Position paper from Scientific Departments from Brazilian Academy of Neurology

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