Antisense-based therapy for the treatment of spinal muscular atrophy
F Rigo, Y Hua, AR Krainer, CF Bennett - 2012 - rupress.org
F Rigo, Y Hua, AR Krainer, CF Bennett
2012•rupress.orgOne of the greatest thrills a biomedical researcher may experience is seeing the product of
many years of dedicated effort finally make its way to the patient. As a team, we have worked
for the past eight years to discover a drug that could treat a devastating childhood
neuromuscular disease, spinal muscular atrophy (SMA). Here, we describe the journey that
has led to a promising drug based on the biology underlying the disease.
many years of dedicated effort finally make its way to the patient. As a team, we have worked
for the past eight years to discover a drug that could treat a devastating childhood
neuromuscular disease, spinal muscular atrophy (SMA). Here, we describe the journey that
has led to a promising drug based on the biology underlying the disease.
One of the greatest thrills a biomedical researcher may experience is seeing the product of many years of dedicated effort finally make its way to the patient. As a team, we have worked for the past eight years to discover a drug that could treat a devastating childhood neuromuscular disease, spinal muscular atrophy (SMA). Here, we describe the journey that has led to a promising drug based on the biology underlying the disease.
rupress.org
