Thomsen and Becker disease

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At a Glance

Live from database

Regulatory Watchboard

6 events
Feb 2026Evaluation of the Role of miR-1 in the Pathogenesis and as a Biomarker in Muscular Dystrophies and Congenital Myopathies

University Hospital, Clermont-Ferrand — NA

TrialRECRUITING
Jul 2025Global Open-Label Extension Study of Del-desiran for the Treatment of DM1

Avidity Biosciences, Inc. — PHASE3

TrialENROLLING BY INVITATION
Mar 2025A Natural History Study of RYR1-Related Disorders

National Institutes of Health Clinical Center (CC)

TrialRECRUITING
Jun 2024Phenotype - Genotype Correlation in a Sample of Egyptian Patients With Congenital Myopathies and Congenital Muscular Dystrophies

Ain Shams University

TrialRECRUITING
May 2024Global Study of Del-desiran for the Treatment of DM1

Avidity Biosciences, Inc. — PHASE3

TrialACTIVE NOT RECRUITING
Jul 2019

OTEZLA®: FDA approved

OTEZLA is indicated for the treatment of adult patients with oral ulcers associated with Beh®et®s Disease.

FDAcompleted

Data from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Financial Assistance

1 program

FINANCIAL LANDSCAPE SUMMARY

1

Total programs

1

Open now

1

Copay cards

Copay Assistance1

OTEZLA�

Amgen Inc.

OpenContact for detailsApply ↗

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Approved Treatments

1 FDA-approved

OTEZLA�

(apremilast)Orphan drug

Amgen Inc.

Phosphodiesterase 4 Inhibitor [EPC]

12.1 Mechanism of Action Apremilast is an oral small molecule inhibitor of phosphodiesterase 4 (PDE4) specific for cyclic adenosine monophosphate (cAM...

Approved Jul 2019FDA label ↗

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Clinical Trial Landscape

2 active trials
2Phase 3
2Total recruiting
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Recent News & Research

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Specialist Network

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