by CheckRare Staff | Aug 9, 2023
Cary Harding, MD, Professor of Molecular and Medical Genetics at Oregon Health & Science University (OHSU), highlights the data evaluating SYNB1934 to treat phenylketonuria (PKU). Uncontrollable levels of the amino acid phenylalanine (Phe) cause PKU,...
by CheckRare Staff | Aug 8, 2023
Scott Schobel, MD, chief medical officer at Vico Therapeutics discussed the latest data testing an antisense therapy to treat Huntington’s disease and spinocerebellar ataxia type 1 (SCA1) and type 3 (SCA3). Huntington’s disease manifests as a rare...
by CheckRare Staff | Aug 7, 2023
Philip L. Pearl, MD, Director, Epilepsy and Clinical Neurophysiology, Boston Children’s Hospital, William G. Lennox Chair and Professor of Neurology, Harvard Medical School, Boston, MA discusses aromatic L-amino acid decarboxylase (AADC) deficiency and the recently...
by CheckRare Staff | Aug 4, 2023
Michael Thorner, MB, Vice President of Endocrine Science at Lumos Pharma discusses the latest research on LUM 201 (ibutamoren) to treat idiopathic pediatric growth hormone deficiency. Growth hormone deficiency is a rare endocrine disorder....
by CheckRare Staff | Aug 3, 2023
Vera Bril, MD, Professor of Medicine at the University of Toronto discusses the recent approval rozanolixizumab to treat myasthenia gravis. Myasthenia gravis is a chronic autoimmune neuromuscular disease characterized by weakness of the skeletal...
