Maze Therapeutics to Present Phase 1 Results
Amicus Therapeutics Announces Presentations and Posters at the 19th Annual WORLDSymposium™...
AskBio has an adeno-associated virus (AAV)-based gene therapy platform and has been driving Bayer’s pipeline of gene therapies that are developed across a range of neuromuscular, central nervous system, cardiovascular and metabolic disease indications. Most advanced in the clinic are...
Babies born in North Carolina will now be screened for two additional disorders, along with the other disorders screened for by the NC Department of Health and Human Services’ Newborn Screening Program. Newborns will now be screened for two Lysosomal Storage Disorders — Mucopolysaccharidosis Type...
Astellas Announces Hold Lifted by FDA on FORTIS Clinical Trial of AT845 Investigational Treatment for Adult Patients with Late-Onset Pompe Disease
Maze planning Phase 2 study of substrate reduction therapy in 2023
Selecta Biosciences and Astellas Announce Exclusive Licensing and Development Agreement for Xork IgG Protease
Recently I met a lovely man named Dave Ross who has a rare disease called Cowden Syndrome. 2 years ago he set up a virtual support group for male patients and caregivers to help support the mental health side of living with a Rare Disease/Disorder. David has just set up additional meetings for...
KEY POINTS In new research, adults with rare disorders discussed their key challenges and how they are combatting them. Lack of awareness among healthcare providers and the public leads to poor medical care and stigma. Many people with rare diseases take it upon themselves to engage in...
Nexviazyme is a 2nd generation Enzyme Replacement Therapy (ERT) for Pompe disease. Genzyme Corporation has put an application in to Medsafe NZ for assessment and approval. Then we need to cross our fingers that PHARMAC will approve it for funding! Nexviazyme status at Medsafe Information on...
Pharmac’s year in review leaves a lot to be desired