by Madaline Spencer | Mar 9, 2026
The US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to nomlabofusp for the treatment of patients with Friedreich’s ataxia. Friedreich’s ataxia is a progressive, inherited condition that affects the nervous system and causes...
by Madaline Spencer and Scott Harwood | Mar 6, 2026
John Taggart, Head of Communications at Niemann-Pick UK (NPUK), discusses the ASMD Perspective Index and its use in diagnosing Niemann-Pick disease. Niemann-Pick disease is an inherited condition characterized by abnormal lipid metabolism that causes harmful...
by Madaline Spencer and Scott Harwood | Mar 5, 2026
Bob Stevens, Group CEO of the MPS Society and Rare Disease Research Partners in the UK, discusses global efforts towards developing treatment options for patients with rare lysosomal storage disorders (LSDs). Mr. Stevens has been involved with the UK MPS...
by Madaline Spencer and Scott Harwood | Mar 4, 2026
Dawn Laney, MS, Genetic Counselor at Emory University School of Medicine, discusses the importance of newborn screening and the need for more effective treatments in infantile Krabbe disease. Krabbe disease is a lysosomal disorder that affects the entire body,...
by Madaline Spencer | Mar 4, 2026
The US Food and Drug Administration (FDA) has granted accelerated approval of Yuviwel (navepegritide; previously TransCon CNP) for the treatment of patients two years of age and older with achondroplasia. Achondroplasia is a disorder of bone growth that prevents the...
