Report highlights disconnect between young patients and caregivers
A new report reveals a communication gap between young people with rare diseases and their caregivers in preparing for adulthood.
News
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New study shows blocking RNA molecule could boost muscle repair in DMD
A mouse study suggests that blocking microRNA-33 may one day help reduce muscle damage in people with Duchenne muscular dystrophy
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Precision BioSciences receives FDA Orphan Drug Designation for PBGENE-DMD
Precision BioSciences has announced that PBGENE-DMD has received FDA Orphan Drug Designation.
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People with DMD want more inclusive sex education, study finds
People with Duchenne muscular dystrophy (DMD) and similar conditions want more inclusive sexual education that meets their unique needs.
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Researchers expand hope for DMD while grappling with setbacks
Advances in gene and small molecule therapies offer new hope for Duchenne muscular dystrophy, but no cure currently exists.
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FDA clears way for Sarepta to restart Elevidys shipments
Patients with DMD who can still walk will regain access to Elevidys as Sarepta resumes shipments following FDA clearance.
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Case report examines acute respiratory distress in a teenage girl with DMD
A recent case report examines the rare case of a teenage girl with DMD that was complicated by acute respiratory distress syndrome.
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Sarepta voluntarily pauses Elevidys shipments in the US
Sarepta Therapeutics has announced that it will voluntarily stop all shipments of the DMD therapeutic agent Elevidys.
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New study identifies simple test to predict fall risk in children with DMD
New research has found that the the Four-Square Step Test can help identify children with DMD at risk of frequent falls
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New study provides insights into beta-blocker therapy efficacy in DMD
Beta-blocker therapy appears to be beneficial in the context of Duchenne muscular dystrophy (DMD) complicated by cardiac dysfunction.
