Batten Disease News

October 23, 2020October 23, 2020

Brineura Now Available Through NHS Scotland to Children With CLN2 Disease

Batten Disease News

The Scottish Medicines Consortium (SMC) has recommended that Brineura (cerliponase alfa) — the first and only approved treatment for late infantile Batten disease, also known as CLN2 disease — be made available ... Read more

October 16, 2020October 16, 2020

CBD Treatment Found Effective for Severe Seizures in Batten Disease Case

Batten Disease News

Cannabinoids were used to successfully treat seizures in a patient with Batten disease for whom conventional therapies alone failed, according to a case report, suggesting that cannabinoids should be investigated ... Read more

October 9, 2020October 9, 2020

First Report Published of Batten and Moyamoya Diseases Occurring in Same Patient

Batten Disease News

A patient with both Batten disease and Moyamoya disease — a condition where blood vessels in the skull become obstructed or narrowed, restricting blood flow to the brain — has ... Read more

October 2, 2020October 2, 2020

Amicus’ Gene Therapy for CLN6 Batten Given PRIME Status in Europe

Batten Disease News

The European Medicines Agency (EMA) has given priority medicines (PRIME) status to AT-GTX-501, Amicus Therapeutics‘ investigational gene therapy for CLN6 Batten disease, otherwise known as variant late infantile neuronal ceroid lipofuscinosis ... Read more

September 25, 2020September 25, 2020

Early Spinal Cord Damage Found in Infantile Batten Disease Mouse Model

Batten Disease News

An examination of the spinal cords of mice that lack the PPT1 protein, the underlying cause of infantile Batten disease, revealed the loss of specific neurons, evidence of inflammation, and ... Read more

September 18, 2020September 18, 2020

New Approach to Correct CLN3 Mutation Shows Promise in Juvenile Batten Disease Mouse Model

Batten Disease News

Treating a juvenile Batten disease mouse model with antisense oligonucleotides to correct its underlying disease-causing mutation led to less waste buildup in the brain, improved motor skills, and longer survival, a ... Read more

September 11, 2020September 11, 2020

Abeona Partners With Taysha to Advance ABO-202 as Potential Gene Therapy

Batten Disease News

Abeona Therapeutics and Taysha Gene Therapies have entered into agreements concerning ABO-202, an investigative gene therapy for infantile Batten disease. The agreements give Taysha worldwide exclusive rights to intellectual property ... Read more

August 28, 2020September 29, 2020

Xperiome Platform Aims to Streamline Searches for Rare Diseases, Gain Patient Input

Batten Disease News

Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient input ... Read more

August 28, 2020August 28, 2020

FDA Names Investigational Therapy BBDF-101 Orphan Drug, Rare Pediatric Disease

Batten Disease News

The U.S. Food and Drug Administration (FDA) has awarded orphan drug designation and rare pediatric disease designation to BBDF-101, an investigational treatment for Batten disease. Theranexus is developing BBDF-101 — ... Read more

August 21, 2020August 27, 2020

EveryLife Foundation Launches Scholarship Fund for Rare Disease Community in US

Batten Disease News

The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to support individuals with rare disorders who are pursuing personal goals through training and education. The ... Read more

August 21, 2020August 21, 2020

Neurogene’s CLN7 Gene Therapy Granted FDA Orphan Drug Designation

Batten Disease News

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to a gene therapy being developed by Neurogene for the treatment of CLN7 Batten disease. This marks the second Neurogene gene ... Read more

August 14, 2020September 15, 2020

New Streaming Channel Showcases Rare Disease Films

Batten Disease News, COVID-19 News

When the COVID-19 pandemic forced the postponement of a rare disease film festival originally slated for May, its organizers set out to find a new way to bring the films ... Read more

July 31, 2020August 3, 2020

URMC Hopes New IDDRC Research Designation Leads to Scientific Advances

Batten Disease News

The University of Rochester Medical Center (URMC) hopes to parlay its new designation as an Intellectual & Developmental Disabilities Research Center (IDDRC) into scientific advances for conditions such as autism ... Read more

July 24, 2020July 24, 2020

Role of CLN6 in Batten Discovered, Shedding Light on Disease

Batten Disease News

The proteins CLN6 and CLN8 — which are deficient in people with two forms of Batten disease — were found to interact directly with each other, forming a functional complex ... Read more

July 17, 2020July 17, 2020

Lymphocyte Vacuolization Might Be Used to Measure Batten Severity

Batten Disease News

The number of fluid-containing cellular compartments called vacuoles within immune cells could help to predict the severity of Batten disease, a new study suggests. The study, “Quantifying lymphocyte vacuolization serves ... Read more