Biogen Inc.: FDA and EMA Accept Applications for Higher Dose Regimen of Nusinersen in SMA
Applications are based on data from the DEVOTE study, which demonstrate the potential for the investigational higher dose regimen of nusinersen to advance the treatment of SMA CAMBRIDGE, Mass., Jan. 2 ...
FDA and EMA Accept Applications for Higher Dose Regimen of Nusinersen in SMA
We are pleased to announce that our applications for the higher dose regimen of nusinersen are now under review in the US and Europe,” said Stephanie Fradette, Pharm.D., Head of the Neuromuscular ...
Too Old to Operate56m
Association of SC GM Atrophy With Clinical Disability in SMA
The following is a summary of “Spinal cord gray matter atrophy is associated with disability in spinal muscular atrophy,” ...
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MRC Holland Gains IVDR Certification for Spinal Muscular Atrophy Newborn Screening Test
The Amsterdam-based biotech firm said that its melt curve analysis-based test is used as a first-tier genetic screening tool to aid the diagnosis of SMA.
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Woman gives birth to healthy child while on Spinraza treatment
A woman with SMA type 3 became pregnant while on Spinraza treatment and gave birth to a healthy child, per a case report.
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'Just a miracle:' Baby with rare genetic condition responds to experimental treatment
Asher Cantrell, diagnosed with SMARD 1, has received experimental gene therapy after social media support and is currently ...
Scholar Rock Sets Sights on $2 Billion Apitegromab Revenue Amid Spinal Muscular Atrophy Market Growth
Scholar Rock aims for a 2025 launch of apitegromab in SMA, expecting $2 billion in global revenue and significant growth from upcoming trials and favorable data.
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Spinal muscular atrophy type 1 in the Caribbean: the first case report from the Dominican Republic
Spinal muscular atrophy (SMA) is a progressive genetic neuromuscular condition ... Neurologically, the patient scores 7 points on the Hammersmith Infant Neurological Examination scale and 3 points on ...
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Dead infant found in Italian baby box after alarm fails to notify priest
The lifeless body of a male infant was found in a box for abandoned babies in southern Italy on Thursday, with the local priest saying an alarm meant to alert him failed to trigger. The baby ...
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Spinal Muscular Atrophy (SMA)
Spinal muscular atrophy, SMA, a genetic neuromuscular disorder with progressive muscle wasting due to mutation in the SMN1 gene, deficiency in SMN protein, and loss of motor neurons, 3D illustration ...
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Spinal Muscular Atrophy (SMA)
Patients with SMA, which has no cure, typically achieve a better quality of life through physical therapy, posture management, and, among other things, nutritional support. 4 Providing individualized ...
Novartis gene therapy helps children with rare muscle disorder in study
Swiss drugmaker Novartis said on Monday its gene therapy helped improve motor function in children with a rare muscle disorder that leaves patients too weak to walk, talk and swallow.