Oligonucleotides,_antisense

Drug Candidate BIO101 for Spinal Muscular Atrophy as Monotherapy or Combined With the Antisense Oligonucleotide ASO-10-27.

<p><b>BACKGROUND</b></p><p>Spinal muscular atrophy (SMA) is a neuromuscular disease caused by loss of survival of motor neuron (SMN) protein inducing progressive muscle weakness and atrophy due to motor neurons degeneration. Despite benefits of SMN restoration therapies in patients, motor defects are...

๐Ÿ—“๏ธ 2025-10-23
๐Ÿ“ฐ Publication: Journal Of Cachexia Sarcopenia And Muscle
Read MoreDrug Candidate BIO101 for Spinal Muscular Atrophy as Monotherapy or Combined With the Antisense Oligonucleotide ASO-10-27.

Subscribe to the SCWD Newsletter

Stay Informed with the Latest Updates and Exclusive Insights!