Posts by: Alice Melao

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

December 12, 2017

FDA Grants PLX-100 Orphan Drug Designation to Treat Batten Disease

News

Polaryx Therapeutics’ investigative drug PLX-100 has been granted orphan drug designation (ODD) by the U.S. Food and Drug Administration (FDA) for the treatment of neuronal ceroid lipofuscinosis (NCL), also ... Read more

July 25, 2017

CLN5 Protein Loss in Batten Disease Led to Fewer Functional Neurons in Mouse Study

News

Researchers studying the loss of the CLN5 protein, which causes Batten disease, found that it leads to the reduced production of new fully functional neurons. It also increased neuronal ... Read more

May 4, 2017August 21, 2017

ABO-201 Improves Inflammation, Movement in Mice with Juvenile Batten Disease, Study Shows

News

Abeona Therapeutics‘ ABO-201 reduced nerve inflammation and improved movement in mice with juvenile Batten disease (JBD), according to a preclinical trial study. Scientists at Abeona designed ABO-201 to overcome the effects ... Read more

Posts by: Alice Melao

Alice Melao

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

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

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

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

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

Early Detection of Retinal Dysfunction May Help Diagnosis

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

FDA Grants PLX-100 Orphan Drug Designation to Treat Batten Disease

CLN5 Protein Loss in Batten Disease Led to Fewer Functional Neurons in Mouse Study

ABO-201 Improves Inflammation, Movement in Mice with Juvenile Batten Disease, Study Shows

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