cln gene – NCL 2012

Infantile batten disease

CLN5 gene therapy trial now recruiting children at New York site

Infantile batten disease

Gene therapy shows promise in mice, supports late infant Batten trial

Infantile batten disease

Amicus halts gene therapy program for CLN6 Batten disease

Juvenile batten disease

CLN6 mutations can lead to Batten without vision loss, 3 cases show

Infantile batten disease

TSHA-118 gene therapy for CLN1 disease to be tested in Canadian trial

Infantile batten disease

CLN1 management guidelines developed by international experts

Adult batten disease

Taysha Gene Therapies Announces the Launch of Clinical Development of TSHA-118 for the Treatment of CLN1 Disease | Nation / World

Infantile batten disease

Taysha Gene Therapies Announces Clinical Development of TSHA-118 for the Treatment of CLN1 Disease

Juvenile batten disease

Batten disease 2021 research grants awarded to 2 scientists in Australia

Infantile batten disease

CLN5 gene therapy trial set to start early next year in Rochester

Infantile batten disease

Late Childhood Gene Therapy Supported by $ 100 Million Funding

Congenital batten disease

New variant of CTSD gene not linked to rare CLN10 latte in adolescent case

Infantile batten disease

FDA clears NGN-101 gene therapy to be tested in patients with CLN5 Batten

Infantile batten disease

Neurogen Natural History Study for Batten Still Enrolling CLN5 Patients

Infantile batten disease

Batten’s Gene Therapy Achieves Orphan Drug and Rare Pediatric Disease Status

Juvenile batten disease

“We pray for a miracle”: 3 Albertan brothers and sisters diagnosed with rare and terminal illness

Congenital batten disease

Exicure Announces Next Neuroscience Pipeline Update During Virtual R&D Day | Business

Congenital batten disease

Exicure Announces Next Neuroscience Pipeline Update During Virtual R&D Day

Infantile batten disease

EMA offers potential gene therapy for CLN5 bat orphan drug status

Infantile batten disease

Neurogene Announces EMA Grant Orphan Drug Designation to CLN5 Batten Disease Gene Therapy

Juvenile batten disease

Gene therapy shows promise in mouse model of CLN8 disease

Juvenile batten disease

“1 billion in 8”: How a devastating genetic defect struck 3 Alberta siblings

Juvenile batten disease

BDSRA Australia Announces Winners of Batten Research Grants 2020

Infantile batten disease

Caregivers of children with CLN5 sought for neurogenic study interviews

Infantile batten disease

Scientist Gets Grant to Study CLN6 Batten and Advance Early Trials

Congenital batten disease

News beyond the pandemic – February 19

Juvenile batten disease

CLN3 gene therapy shows safety, possible efficacy in trial: Amicus

Juvenile batten disease

Research sheds light on vision loss in Batten disease

Juvenile batten disease

Amicus Therapeutics announces first positive clinical study

Juvenile batten disease

Amicus Therapeutics Announces Positive Initial Clinical Results

Congenital batten disease

New research sheds light on vision loss in Batten disease – sciencedaily

Infantile batten disease

Neurogene partners with university to advance gene therapy technologies

Congenital batten disease

Exicure Announces Agenda for Next Neuroscience Pipeline Update During Virtual R&D Day

Juvenile batten disease

Cannabis oil as an adjunct to relieve seizures in CLN6 latte

Infantile batten disease

Neurogene Adds Additional $ 115 Million To Fund For Gene Therapy Trials

Juvenile batten disease

In CLN6 Batten, AT-GTX-501 effective after 2 years, interim results show

Infantile batten disease

Neurology today

Infantile batten disease

Amicus Therapeutics Announces Additional Positive Interim Clinical Data for CLN6 Batten Disease Gene Therapy at Child Neurology Society 49th Annual Meeting

Infantile batten disease

Amicus Therapeutics Announces Positive Interim News

Juvenile batten disease

Amicus Gene Therapy for CLN6 Batten Receives PRIME Status in Europe

Juvenile batten disease

New approach to treat CLN3 disease shows promise in mouse model

Infantile batten disease

CLN7 Batten Gene Therapy Obtains Orphan Drug Designation From FDA

Juvenile batten disease

County Durham mother opens up about heartbreaking struggle after son diagnosed with incurable genetic disease

Juvenile batten disease

Role of CLN6 in Batten discovered, shedding light on lysosomal disease

Juvenile batten disease

Lymphocyte vacuolation could be used to measure the severity of Batten

Infantile batten disease

Gene therapy for CLN5, a form of late infantile batten, named orphan drug by the FDA

Infantile batten disease

Pakistani study describes new mutations that cause CLN5 disease

Juvenile batten disease

Amicus’ CLN3 Batten Disease Gene Therapy Obtains FDA Fast Track Status

Infantile batten disease

FDA Promotes Clinical Trial of Potential Treatment for Juvenile Lattes, PLX-200

Juvenile batten disease

Polaryx Therapeutics Receives FDA IND Approval to Study PLX-200 Treatment for Patients with Juvenile Neuronal Ceroid Lipofuscinosis

Infantile batten disease

Critical period of vision degeneration in CLN2 patients aged 4 to 7 years

Juvenile batten disease

Study sheds light on CLN3’s involvement in juvenile batten disease

Infantile batten disease

Researchers discover new mutations linked to late childhood Batten disease

Juvenile batten disease

Vision loss in children could mean juvenile slat disease, study finds

Infantile batten disease

Gene Therapy for CLN2 Batten Shows Good Safety in Small Clinical Trial

Juvenile batten disease

Juvenile Batten Mouse Study Finds Fat Molecule Improves Longevity, Facilitates Neuron Loss

Adult batten disease

Mutant Sheep Raised In Lab To Fight Fatal Childhood Brain Disease | Gene modification

Infantile batten disease

Patients with signs of LINCL should be tested for CLN5 / CLN8 defects

Juvenile batten disease

Scientists discover new mutation in CLN3 associated with JNCL

Congenital batten disease

“The worst day of our lives” – CBS Denver

Congenital batten disease

Batten disease research advancing at substantial rate, review suggests

Juvenile batten disease

Alpha-synuclein may contribute to the progression of Batten disease

Juvenile batten disease

CLN3 deletion affects genes responsible for ganglioside production, study finds

Infantile batten disease

Compounds similar to flupirtine show promise for treatment of Batten disease, study reports – Batten Disease News

Infantile batten disease

Twins with Rare Batten Disease Challenge the Chances of Reaching Teens

Infantile batten disease

Batten study uses CT scans to monitor brain alterations in sheep models

Infantile batten disease

Natural CLN7 pattern found in type of monkey may aid research, study finds

Juvenile batten disease

Previously unknown role for Batten disease protein identified

Juvenile batten disease

Safe, but ineffective, CellCept in Phase 2 clinical trial for CLN3 disease

Infantile batten disease

Researchers identify 5 new mutations linked to the form of Batten disease

Infantile batten disease

New mouse model of late infantile Batten disease may aid the quest for therapy

Infantile batten disease

Batten study describes early signs and progression of CLN5 disease

Congenital batten disease

Genome sequencing quickly diagnosed CLN8 disease in Chinese boy

Infantile batten disease

Vision loss in Batten disease prevented by gene therapy in mouse study

Juvenile batten disease

Batten Disease Kills 3 Utah Siblings In 3 Days

Infantile batten disease

2 Batten disease clinical trials recruit patients to test gene therapies

Infantile batten disease

Batten disease trial funded by the Charlotte and Gwenyth Gray Foundation

Juvenile batten disease

Potential gene therapy for juvenile latte disease named orphan drug by FDA

Infantile batten disease

In mice with Batten LINCL disorder, Lopid treatment increases longevity

Juvenile batten disease

Batten’s disease movement improves with PDE4 enzyme inhibitors, study finds

© 2022 NCL 2012.