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Newborn Screening for SMA Appears to Offer Functional Benefits

German cohort study suggests earlier initiation of treatment benefits sitting, walking

Switzerland , Montreal , Quebec , Canada , Germany , Austria , German , Jennifer-henderson , Maryam-oskoui , Janbernd-kirschner , University-of-freiburg , Medpage-today

Givinostat Slows Duchenne Muscular Dystrophy Decline

Trial details the results that led to recent FDA approval

Rome , Lazio , Italy , Germany , Eugenio-mercuri , Givinostat-duvyzat , Janbernd-kirschner , Sophie-putka , University-of-freiburg , Universita-cattolica-del-sacro-cuore , North-star-ambulatory-assessment , Collaborative-trajectory-analysis-project

Study supports daily steroid doses for children with Duchenne muscular dystrophy

New research published in JAMA recommends daily steroid doses for children with Duchenne muscular dystrophy (DMD), marking a significant change in how the disease is treated.

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New study expands evaluation of gene therapy for spinal muscular atrophy

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Amsterdam, April 6, 2021 - The rarity of spinal muscular atrophy (SMA) means that promising new treatments may be tested in only a limited spectrum of patients before approval. Investigators evaluated a newly approved drug, onasemnogene abeparvovec, in a broader spectrum of patients in order to obtain expanded data on its side effects profile. They report in the
Journal of Neuromuscular Diseases that the drug is associated with an immune response against the adeno-associated viral vector and needs careful monitoring, but showed no long-term adverse effects.
In recent years, the availability of a growing number of drug treatments has significantly changed the course of the SMA. One of these is onasemnogene abeparvovec (Zolgensma®), an adeno-associated viral (AAV9) vector-based gene therapy that introduces a functional copy of the

Germany , Bonn , Nordrhein-westfalen , Amsterdam , Noord-holland , Netherlands , Janbernd-kirschner , Drug-administration , European-medicine-agency , Department-of-neuropediatrics , University-hospital-bonn , Neuromuscular-diseases

Researchers evaluate new drug for spinal muscular atrophy in a broader spectrum of patients

Researchers evaluate new drug for spinal muscular atrophy in a broader spectrum of patients
The rarity of spinal muscular atrophy (SMA) means that promising new treatments may be tested in only a limited spectrum of patients before approval. Investigators evaluated a newly approved drug, onasemnogene abeparvovec, in a broader spectrum of patients in order to obtain expanded data on its side effects profile. They report in the
Journal of Neuromuscular Diseases that the drug is associated with an immune response against the adeno-associated viral vector and needs careful monitoring, but showed no long-term adverse effects.
In recent years, the availability of a growing number of drug treatments has significantly changed the course of the SMA. One of these is onasemnogene abeparvovec (Zolgensma®), an adeno-associated viral (AAV9) vector-based gene therapy that introduces a functional copy of the

Germany , Bonn , Nordrhein-westfalen , Janbernd-kirschner , Emily-henderson , Drug-administration , European-medicine-agency , Department-of-neuropediatrics , University-hospital-bonn , Neuromuscular-diseases , ஜெர்மனி , பொன்

'SMA Patients No Longer Doomed If Diagnosed Early'

'SMA Patients No Longer Doomed If Diagnosed Early'
It is necessary to correctly evaluate which children can be treated with the approved drugs, says Dr. Soyhan Bagci.
Gene therapy for spinal muscular atrophy (SMA) patients is effective for infants whose symptoms have never started or have just started, according to a pediatric and neonatal intensive care physician.
"After gene therapy is applied to children who are connected to a respiratory device and cannot walk, it is unfortunately not possible for them to leave the breathing apparatus, start running and return to normal life," Dr. Soyhan Bagci from Rheinische Friedrich-Wilhelms University in Bonn, Germany told Anadolu Agency.

Germany , Anadolu , Turkey-general , Turkey , Bonn , Nordrhein-westfalen , Soyhan-bagci , Janbernd-kirschner , Rheinische-friedrich-wilhelms-university-in-bonn , Anadolu-agency , University-hospital-bonn , Rheinische-friedrich-wilhelms-university