Spinraza

May 19, 2025

The approval of three spinal muscular atrophy therapies—SPINRAZA (Ionis/Biogen), EVRYSDI (Roche), and the groundbreaking gene therapy ZOLGENSMA (Novartis) has significantly advanced treatment options for a condition that previously had limited solutions. These spinal muscular atrophy (SMA) therapies offer patients ...

May 12, 2025

Spinal muscular atrophy (SMA) is the second most prevalent autosomal recessive disorder, with a carrier frequency of approximately 1 in 40 to 50 individuals and an estimated occurrence of 1 in 10,000 live births. The majority of SMA cases are caused by the deletion of a portion of the SMN1 gene, which impairs the p...

May 06, 2019

SMA (Spinal Muscular Atrophy) - a disease that takes away the physical strength of people by affecting the motor nerve cells in the spinal cord, also people have a problem in walking, eating, or breathing. Spinal Muscular Atrophy market size is increasing at a CAGR of 10.42% for the study period (2017-2028). ...

Apr 29, 2019

Spinal muscular atrophy (SMA) being a rare genetic neuromuscular disorder results in progressive muscle weakness and atrophy. It affects the part of the nervous system that controls voluntary muscle movement. Spinal Muscular Atrophy Current therapeutic management revolves around supportive and multidisciplinary car...

Apr 24, 2019

Spinal muscular atrophy is a genetic disorder characterised by weakness and wasting (atrophy) of skeletal muscles, used for movement. Loss of specialised nerve cells, called motor neurons which controls muscle movement, results in weakness of muscles. It mostly affects proximal muscles and respiratory muscles. SMA ...

Dec 27, 2018

Vedanta raises USD 27 Million to move microbiome assets through the clinic Vedanta Biosciences raises USD 27 million in its series C round. The cash will bolster its microbiome-derived drugs pipeline. The capital will aid a phase 1/2 trial of VE416 in food allergy, a phase 2 study of VE303 in recurrent...