by Madaline Spencer and James Radke, PhD | May 11, 2023
Christian Marsolais, Ph.D., Chief Medical Officer of Theratechnologies, discusses three studies presented at the American Association for Cancer Research (AACR) conference targeting SORT1-positive cancers. Sudocetaxel zendusortide (TH1902) is a proprietary...
by Madaline Spencer | May 5, 2023
Dean Suhr, of the MLD Foundation, provides an overview of the latest clinical milestones in diagnosing and treating metachromatic leukodystrophy (MLD). MLD is a lysosomal disorder due to a deficiency in the enzyme arylsulfatase A (ARSA). It is characterized by...
by Madaline Spencer | May 1, 2023
Shawn Connor, father of a girl with Rett syndrome, talks about the advocacy group, Believe in Brynn. As Mr. Connor explains, Believe in Brynn is a patient advocacy group focused on the education of Rett syndrome and funding clinical research. The group hosts...
by Madaline Spencer | Apr 25, 2023
Shawn Connor, father of a girl with Rett syndrome, talks about his daughter’s diagnostic journey. Rett syndrome is a rare, progressive neurodevelopmental condition that primarily affects girls. These girls appear to develop normally during the first 6 to 18...
by Madaline Spencer | Apr 25, 2023
Sitra Tauscher-Wisniewski, MD, Vice President of Clinical Development & Analytics at Novartis Gene Therapies, discusses the importance of early diagnosis of spinal muscular atrophy (SMA). SMA is a genetic disease that affects motor neurons in the...
