Three years of treatment with Brineura (cerliponase alfa) was seen to effectively and durably delay the progression of late infantile Batten…
Articles by Alice Melao
High levels of a protein associated with Parkinson’s disease, called alpha-synuclein, may contribute to the progression of late infantile…
Mutated forms of CLN3 protein may be involved in the abnormal activation of signals in brain cells that, in excess,…
Amicus Therapeutics is recruiting children for its new Phase 1/2 clinical trial that will assess the safety and efficacy…
Abeona Therapeutics has acquired the global rights to REGENXBIO’s proprietary NAV AAV9 delivery system to develop gene therapies for…
Amicus Therapeutics has gained global development and commercial rights to 10 gene therapy programs for lysosomal storage disorders, including three…
A new system of vagus nerve stimulation (VNS) therapy was implanted in the first patient with drug-resistant epilepsy as part of…
ABO-202, Abeona Therapeutics‘ investigational candidate for the treatment of infantile Batten disease has received orphan drug status by the…
Early detection of abnormalities to the eye’s retina and loss of visual acuity may help diagnose neuronal ceroid lipofuscinosis (NCL),…
A new bioinformatic tool called Aminode, developed to help researchers predict the potential outcome of genetic mutations, found that most…