Patients are continuing to be treated in clinical trials testing Abeona Therapeutics‘ investigational gene therapies — ABO-101 for those with …
Articles by Patricia Inácio, PhD
The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to Seelos Therapeutics‘ SLS-005 (trehalose) to treat Sanfilippo…
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Seelos Therapeutics‘ SLS-005 (trehalose) to treat…
Orchard Therapeutics announced the dosing of a first patient in a clinical trial evaluating its investigational cell-based gene…
The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However,…
Screening for metabolic disorders, such as Sanfilippo syndrome, should be considered in patients initially diagnosed with Landau Kleffner…
Accumulation of the protein alpha-synuclein in the brain — a hallmark of neurodegenerative diseases like Parkinson’s — does not factor…
Researchers at the National Institutes of Health have developed an induced pluripotent stem cell (iPSC) line from a 1-year-old…
Pentosan polysulfate sodium, an approved medication commonly used to treat bladder pain, will now be repurposed to potentially treat mucopolysaccharidosis (MPS) under the…
Phoenix Nest has acquired the exclusive rights from the University of Manchester for the development of a gene therapy to…