by Madaline Spencer | May 12, 2023
Ponni Subbiah, MD, MPH, Chief Medical Officer at Acadia Pharmaceuticals, discusses the results of the LAVENDER trial, which led to the approval of trofinetide (DAYBUE) in patients with Rett syndrome. Rett Syndrom Background Rett syndrome is a rare...
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...
