On the occasion of this Rare Disease Day, Mstone Partners Healthcare and our portfolio companies are proud to stand with the ...

Please provide your email address to receive an email when new articles are posted on . The FDA has approved an adeno-associated virus vector-based gene therapy to treat both adult and pediatric ...

Aromatic amino acid decarboxylase (AADC) deficiency is a rare, autosomal recessively inherited neurometabolic disorder characterised by markedly reduced synthesis of key monoamine neurotransmitters ...