MRC Holland Announces Quick and Low-Cost Blood Test to Screen Newborns for SMA
MRC Holland has launched a newborn screening test, called SALSA MC002 SMA Newborn Screen, that uses a blood sample to…
Articles by José Lopes, PhD
Phase 1 Trial of AVXS-101 in SMA Type 2 Fully Enrolled
Patient enrollment is complete for AveXis’ Phase 1 clinical trial testing its gene therapy candidate AVXS-101 in patients with spinal…
AVXS-101 May Be ‘Transformative’ Breakthrough for SMA Type 1, Cure SMA President Says
AveXis‘ gene therapy AVXS-101, now under review for approval to treat infants with spinal muscular atrophy (SMA) type…
ICER to Compare Clinical Efficacy and Value of Spinraza and AVXS-101 in Treating SMA
The Institute for Clinical and Economic Review (ICER) will compare the clinical effectiveness and value of the only approved…
SMA Added to List of Recommended Screenings for Disease Given to Newborns in US
Spinal muscular atrophy (SMA) has been added to the Recommended Uniform Screening Panel (RUSP) for newborns in the…
#CureSMA2018 – Exicure’s SNA Formulation of Nusinersen Raises SMN Protein Levels with Lesser Toxicity, Mouse Study Reports
Exicure’s gene c prolongs survival, increases the amount of SMN proteins, and shows limited toxicity in a mouse model of…
Decreased Levels of Specific Enzyme Involved in SMA Development in Mouse Study
Decreased levels of an enzyme lead to cellular dysfunction and neuronal degeneration involved in the development of spinal muscular atrophy (SMA), a…
Roche Stops Work on Olesoxime After Disappointing Long-term Results in Phase 2 Trial
Roche has stopped the development of olesoxime, a treatment candidate for spinal muscular atrophy (SMA) following disappointing 18-month…
Phase 1 Trial of SRK-015, Potential SMA Therapy, Completes 1st Dosing, Scholar Rock Reports
Scholar Rock announced it has completed dosing a first group of healthy volunteers in its Phase 1 clinical study of SRK-015, a…
Patients with SMA Types 2 and 3 at Risk of Malnutrition, Altered Metabolism, Study Reports
Patients with spinal muscular atrophy (SMA) types 2 and 3 are at risk of malnutrition, altered metabolic…