Posts by: Alice Melao

Alice Melao

April 5, 2019April 5, 2019

Brineura Over 3 Years Seen to Delay Loss of Motor-Language Skills in CLN2 Children, Trial Data Shows

News

Three years of treatment with Brineura (cerliponase alfa) was seen to effectively and durably delay the progression of late infantile Batten disease, compared to its natural course, in an ongoing and long-term extension study, BioMarin announced. ... Read more

March 22, 2019March 22, 2019

Alpha-Synuclein Protein May Contribute Toward Batten Disease Progression

News

High levels of a protein associated with Parkinson’s disease, called alpha-synuclein, may contribute to the progression of late infantile Batten disease, according to a recent study. The study, “Autophagy–lysosome ... Read more

January 25, 2019January 25, 2019

Mutated CLN3 Protein May Indirectly Promote Abnormal Signaling Cascades Involved in Juvenile Batten Disease, Study Finds

News

Mutated forms of CLN3 protein may be involved in the abnormal activation of signals in brain cells that, in excess, could promote cell death, a study has found, which ... Read more

January 4, 2019January 4, 2019

Amicus Therapeutics Recruiting Children with CLN3 Batten Disease for Gene Therapy Trial

News

Amicus Therapeutics is recruiting children for its new Phase 1/2 clinical trial that will assess the safety and efficacy of an investigational gene therapy for Batten disease caused by ... Read more

November 13, 2018November 13, 2018

Abeona Acquires Rights to Develop Gene Therapies Using NAV AAV9 Gene Delivery System

News

Abeona Therapeutics has acquired the global rights to REGENXBIO’s proprietary NAV AAV9 delivery system to develop gene therapies for Sanfilippo syndrome type A (MPS IIIA) and type B (MPS IIIB). It ... Read more

September 28, 2018September 28, 2018

Amicus Therapeutics’ Expanded Portfolio Includes Potential Gene Therapies for Batten Disease

News

Amicus Therapeutics has gained global development and commercial rights to 10 gene therapy programs for lysosomal storage disorders, including three potential first-to-market therapies for Batten disease. This pipeline expansion resulted from ... Read more

September 11, 2018September 11, 2018

LivaNova Launches Study to Assess VNS Therapy in Drug-resistant Epilepsy Patients

News

A new system of vagus nerve stimulation (VNS) therapy was implanted in the first patient with drug-resistant epilepsy as part of a global registry launched by LivaNova to evaluate the treatment in a real-world ... Read more

April 27, 2018April 27, 2018

ABO-202 Earns EMA’s Orphan Drug Status for Infantile Batten Disease

News

ABO-202, Abeona Therapeutics‘ investigational candidate for the treatment of infantile Batten disease has received orphan drug status by the European Medicines Agency. This new status adds to the previously ... Read more

February 1, 2018

Early Detection of Retinal Dysfunction May Help Diagnosis

News

Early detection of abnormalities to the eye’s retina and loss of visual acuity may help diagnose neuronal ceroid lipofuscinosis (NCL), aka Batten disease, and improve patient care, Brazilian researchers ... Read more

January 23, 2018

Bioinformatic Tool Finds 71% of Batten-causing Mutations in Key Protein Regions

News

A new bioinformatic tool called Aminode, developed to help researchers predict the potential outcome of genetic mutations, found that most disease-causing mutations occur in regions of proteins essential to their ... Read more