Batten Disease News

January 8, 2021January 8, 2021

Taysha, Catalent Partner to Advance Gene Therapies for Batten, Other Disorders

Batten Disease News

Taysha Gene Therapies announced that it has joined with Catalent, a pharmaceutical services provider, to support production of its potential gene therapies for disorders that include CLN1 Batten disease and Rett syndrome. ... Read more

December 18, 2020December 18, 2020

Cannabis Oil as Add-on Therapy Seen to Ease Severe Seizures in CLN6 Batten

Batten Disease News

Adding cannabis oil to standard anti-seizure medications may be an effective way of managing treatment-resistant status epilepticus — life-threatening seizures that require urgent medical treatment — in people with CLN6 ... Read more

December 11, 2020December 11, 2020

Enrollment Begins in Natural History Study of 2 Batten Disease Subtypes

Batten Disease News, COVID-19 News

Neurogene has enrolled the first patient in a natural history study of CLN5 and CLN7 disease, two forms of late-infantile Batten disease, the company announced. The study (NCT03822650), which is still ... Read more

December 4, 2020January 5, 2021

NORD Push for Rare Disease Advisory Councils Focus of Dec. 16 Public Meeting

Batten Disease News

To empower and equip members of the rare disease community to engage state leaders in matters of importance to patients and their families, the National Organization for Rare Disorders (NORD) ... Read more

December 4, 2020December 4, 2020

Juvenile Batten Disease Treatment BBDF-101 Earns FDA’s OK for Pre-Clinical Plan

Batten Disease News

The U.S. Food and Drug Administration (FDA) has given a favorable opinion of the continued preclinical development plan for BBDF-101, a potential new treatment for juvenile Batten disease (CLN3). The plan, made ... Read more

November 26, 2020December 28, 2020

Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe

Batten Disease News, COVID-19 News

The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their health and well-being, according to a ... Read more

November 20, 2020November 20, 2020

BDFA Seeks Funds for Potential Brineura Study to Try Saving Children’s Sight

Batten Disease News

The Batten Disease Family Association (BDFA) is moving forward with a fundraising campaign aimed at developing a United Kingdom study that would use Brineura (cerliponase), an enzyme replacement therapy (ERT), to ... Read more

November 13, 2020November 13, 2020

Taysha, Invitae Partner on Rapid Genetic Testing for Lysosomal, Epileptic Disorders

Batten Disease News

Taysha Gene Therapies has announced a partnership with Invitae to support two of Invitae’s programs that enable more rapid diagnoses for patients with lysosomal storage disorders and epileptic conditions, including ... Read more

November 6, 2020November 6, 2020

Amicus Gene Therapy Effective After 2 Years in CLN6 Batten Children, Interim Results Show

Batten Disease News

Amicus Therapeutics‘ investigational gene therapy AT-GTX-501 stabilized motor and language function in children with variant late infantile neuronal ceroid lipofuscinosis 6 disease — also known as CLN6 Batten disease — two ... Read more

October 30, 2020October 30, 2020

UK Organization ‘Same But Different’ Announces ‘Glimmer of Hope’ Photo Contest Winner

Batten Disease News

A photo of a bespectacled young boy, his red baseball cap slightly askew as he  enjoys time outside, will be featured on the front cover of an upcoming calendar in ... Read more

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, 2020November 10, 2020

Same But Different Asks Public to Vote on ‘Glimmer of Hope’ Calendar Photos

Batten Disease News, COVID-19 News

Same But Different, a nonprofit U.K. group that uses art for social change, is inviting people to choose their favorite photographs in a calendar contest to heighten awareness of rare diseases, ... 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