Spinal muscular atrophy (SMA) encompasses a group of autosomal recessively inherited degenerative neuromuscular disorders. They range in severity from neonatal onset with rapidly progressive weakness and early mortality (SMA-1)...
Venous thromboembolism (VTE) encompasses deep-vein thrombosis (DVT) and pulmonary embolism. VTE is the leading cause of lost disability-adjusted life years and the third leading cause of cardiovascular death in the world.
Anthos Therapeutics, announced the initiation of recruitment for its second Phase III clinical trial investigating abelacimab, its novel dual-acting fully human monoclonal antibody targeting both Factor XI and Factor XIa
Spinal muscular atrophy (SMA) is one of the leading genetic diseases of children and infants. SMA is caused by deletions or mutations of Survival Motor Neuron 1 (SMN1) gene. SMN2, a nearly identical copy of SMN1, cannot...
Humans have two nearly identical copies of the Survival Motor Neuron (SMN) gene, SMN1 and SMN2. In spinal muscular atrophy (SMA), SMN2 is not able to compensate for the loss of SMN1 due to exclusion of...
Bayer announced the start of a Phase III clinical development program “OCEANIC” to investigate the efficacy and safety of asundexian, an oral Factor XIa (FXIa) inhibitor, as a potential new treatment in patients with atrial fibrillation and in patients with a non-cardioembolic ischemic stroke or high-risk transient ischemic attack
No disease-slowing treatment exists for Huntington's disease, but its monogenic inheritance makes it an appealing candidate for the development of therapies targeting processes close to its genetic cause.
GlaxoSmithKline (GSK) and Prosensa announced that the Phase III clinical study of drisapersen, an investigational antisense oligonucleotide, for the treatment...
Sarepta Therapeutics, Inc., announced that the FDA has approved Vyondys 53 (golodirsen). Vyondys 53 is an antisense oligonucleotide from Sarepta�s...
Fresenius Kabi announced it has received FDA 510(k) clearance for the Aurora Xi Plasmapheresis System. The Aurora Xi Plasmapheresis System,...