Batten Disease – Patient Worthy

source: shutterstock.com

Experimental Gene Therapy Showed Promising Safety and Efficacy in Primate Study of Late-Infantile Batten Disease

Post author:Jessica Lynn
Post published:March 5, 2024
Post category:Batten Disease

Children with late-infantile onset Batten disease (CLN2) may be treated with an enzyme replacement therapy (ERT). However, this therapy cannot reverse the damage or progression children have already experienced. Researchers…

geralt / Pixabay

Auditory Sensory Memory System Decline: A Potential Neuromarker for Juvenile-Onset Batten Disease

Post author:Jessica Lynn
Post published:February 20, 2024
Post category:Batten Disease

In total, there are thirteen subtypes of Batten disease. Batten disease, also known as neuronal ceroid lipofuscinosis (NCL), is a rare and inherited neurodegenerative disorder that causes visual impairment, behavioral…

source: pixabay.com

Family Learns of Daughter’s Batten Disease After Routine Eye Exam

Post author:Jessica Lynn
Post published:October 17, 2023
Post category:Batten Disease

7-year-old Isla Edwards suddenly found herself struggling to see. Her vision became fuzzy when trying to focus on objects at a distance. Her parents assumed this meant Isla needed…

Source: Pixabay.com

4th Annual Michael’s Miracle Golf Fundraiser Supports Son with Batten Disease

Post author:Jessica Lynn
Post published:September 26, 2023
Post category:Batten Disease

When Tom and Tammy Parteleno first learned that their son Michael had Batten disease, it felt overwhelming. Scary, even. What would the future bring for their son? The Parteleno family…

source: shutterstock.com

This Town is Showing Support for a Girl with Batten Disease

Post author:James Moore
Post published:June 5, 2023
Post category:Batten Disease

According to a story from CBS News Boston, Scarlett is just six years old and was recently hit with a devastating diagnosis: Batten disease. Unfortunately, very little can be done…

Source: www.pixabay.com

Boy with Batten Disease Pens Book About Marshmallow, His Cane

Post author:Jessica Lynn
Post published:May 25, 2023
Post category:Batten Disease

7-year-old Peter Heath loves to tell jokes, play with Legos, hang out with his friends, and spend time outside. Guided by his white cane, which Peter aptly named Marshmallow, Peter…

Photo by Towfiqu barbhuiya on Unsplash

Enrollment is Complete for a Phase ½ Study on Batten-1 for Batten Disease

Post author:Jessica Lynn
Post published:September 30, 2022
Post category:Batten Disease

According to an article in Batten Disease News, enrollment is complete for a Phase 1/2 clinical trial. Within the trial, researchers will be evaluating Batten-1 for adolescents and adults with…

source: shutterstock

Parents Develop Drugs to Help Their Children with Rare Diseases

Post author:Jessica Lynn
Post published:September 1, 2022
Post category:Rare Disease

Unfortunately, the drug development process can be difficult, especially in the rare disease sphere. Many drug developers choose to focus on larger disease states, as these are typically more…

source: pixabay.com

Treatment Brings Hope to Family of Boy with Batten Disease

Post author:Jessica Lynn
Post published:May 26, 2022
Post category:Batten Disease

For the first three years of Brody Koslowski's life, he seemed - on the surface - to be a healthy, happy boy. So his parents, Matt and Molly, were…

Five Year Old Holly Was Diagnosed With Batten Disease

Post author:Rose Duesterwald
Post published:March 21, 2022
Post category:Batten Disease

Amanda Gilpin, a resident of Karratha in Western Australia, noticed that her little daughter occasionally would roll her eyes in response to some of her motherly advice. At times, she…

A New Collaboration Could Bring A New CLN2 Batten Disease Treatment To Patients Faster

Post author:Trudy Horsting
Post published:November 3, 2021
Post category:Batten Disease

LEXEO Therapeutics and FUJIFILM Diosynth Biotechnologies have just announced their collaborative partnership. The aim is to develop and ultimately manufacture the AAV-mediated gene therapies developed by LEXEO. LEXEO LEXEO is…

source: pixabay.com

LX1004 for CLN2 Batten Disease Earns Rare Pediatric Disease Designation

Post author:Jessica Lynn
Post published:August 3, 2021
Post category:Batten Disease

According to a press release from late July 2021, LX1004, a treatment for patients with CLN2 Batten disease, received both Orphan Drug and Rare Pediatric Disease designations from the…

PublicDomainPictures / Pixabay

hCLN5 Gene Therapy for CLN5 Batten Disease Granted EMA Orphan Drug Status

Post author:Jessica Lynn
Post published:June 29, 2021
Post category:Batten Disease

In a news release from June 29, 2021, biotechnology company Neurogene Inc. shared that its adeno-associated virus (AAV) encoding a codon-optimized human CLN5 transgene (hCLN5) received Orphan Drug designation from…

source: pixabay.com

Two Fathers of Daughters with Batten Disease Created a Bill Which Would Fund Genetic Testing

Post author:Trudy Horsting
Post published:March 8, 2021
Post category:Batten Disease

Mike Jackson's young daughter Evelyn was diagnosed with Batten disease after a neurologist suggested a genetic test which the family could take for free. John Barclay's daughter River passed away…

A Batten’s Disease Flair Up Took Their Christmas Spirit. Their Small Town Gave It Back.

Post author:Sunniva Bean
Post published:December 17, 2020
Post category:Batten Disease

As originally reported in the BBC; In Leyland, Lancashire, Mother Melanie Moffatt describes feeling miserable on Christmas. She was spending it alone with her 10 year old daughter Matilda who…

source: pixabay.com

Positive Data Announced for CLN6 Batten Disease Gene Therapy AT-GTX-501

Post author:Jessica Lynn
Post published:October 15, 2020
Post category:Batten Disease

In recent years, there have been huge progressions in the field of gene therapy. At its core, gene therapy addresses genetic disorders by using genes to directly treat or…

source: pixabay.com

Discovering CNL6’s Role in Batten Disease

Post author:Kendall Mason
Post published:July 7, 2020
Post category:Batten Disease

There are multiple forms of Batten disease, each with their own distinct genetic cause. One of these forms is caused by a mutated CNL6 gene, but researchers were unsure as…

source: pixabay.com

Free Genetic Testing for Lysosomal Storage Diseases Provided by Invitae, Axovant

Post author:Jessica Lynn
Post published:May 14, 2020
Post category:Batten Disease Fabry Disease Pompe Disease Rare Disease Sanfilippo Syndrome

According to Batten Disease News, Invitae and Axovant Gene Therapies partnered up to offer free genetic testing in North America. This will focus on children who are thought to…

Sourced from Pixabay

Is It Safe to Anesthetize Young Patients with Batten Disease?

Post author:Jessica Lynn
Post published:April 15, 2020
Post category:Batten Disease

How will this affect my child? Will this diagnosis change the way my child can receive medical treatment? If an emergency arises, do I have the knowledge needed to…

source: pixabay.com

It Just Makes “Antisense:” Using Genetic Medicine to Treat Rare Diseases

Post author:Jessica Lynn
Post published:March 31, 2020
Post category:Batten Disease Duchenne Muscular Dystrophy Muscular Dystrophy Spinal Muscular Atrophy

As previously published in Scientific American, slightly after her first birthday, Emma Larson lost the use of her legs and started experiencing difficulty crawling. She was diagnosed with spinal muscular…

source: pixabay.com

Faulty Proteins and Failed Genes: Understanding the Biology Behind Congenital Diseases

Post author:Jessica Lynn
Post published:March 23, 2020
Post category:Batten Disease Duchenne Muscular Dystrophy Erythromelalgia Familial Hypercholesterolemia Spinal Muscular Atrophy

Patients with congenital diseases often experience a range of uncomfortable symptoms. On one side of the world, a family with erythromelalgia, a painful condition which can cause redness and swelling…

source: pixabay.com

How Batten Disease has Affected the Carroll Family

Post author:Kendall Mason
Post published:February 28, 2020
Post category:Batten Disease

The Carroll family recently welcomed their daughter Renesmee into the world. They immediately checked for symptoms of Batten disease, a rare, fatal nervous system disorder, as two of Renesmee's siblings…

source: pixabay.com

Neurogene and Behind the Seizure Work to Provide Better Diagnostics for Batten Disease

Post author:Kendall Mason
Post published:February 18, 2020
Post category:Batten Disease

Neurogene, which works to provide treatments for rare neurological disorders, has recently joined with Behind the Seizure. Behind the Seizure was established by two other companies, BioMarin and Invitae, with…

Rare Diseases: A Catch 22 Dilemma

Post author:Rose Duesterwald
Post published:October 28, 2019
Post category:Batten Disease Chagas disease Ebola Fragile X Syndrome Pitt-Hopkins Syndrome Spinal Muscular Atrophy

A guest speaker at the 2019 Rare Disease Forum recently held at the North Carolina Biotechnology Center acknowledged that screening is necessary to justify the need for a particular…

DarkoStojanovic / Pixabay

A Girl Diagnosed with Batten Disease got Treated with a Drug Developed Specifically for Her

Post author:James Moore
Post published:October 21, 2019
Post category:Batten Disease

According to a story from Technology Review, the story of Mila Makovec shows the potential impact that personalized medicine could have on patients while also revealing some of its limitations.…

Get Involved

Experimental Gene Therapy Showed Promising Safety and Efficacy in Primate Study of Late-Infantile Batten Disease

Auditory Sensory Memory System Decline: A Potential Neuromarker for Juvenile-Onset Batten Disease

Family Learns of Daughter’s Batten Disease After Routine Eye Exam

4th Annual Michael’s Miracle Golf Fundraiser Supports Son with Batten Disease

This Town is Showing Support for a Girl with Batten Disease

Boy with Batten Disease Pens Book About Marshmallow, His Cane

Enrollment is Complete for a Phase ½ Study on Batten-1 for Batten Disease

Parents Develop Drugs to Help Their Children with Rare Diseases

Treatment Brings Hope to Family of Boy with Batten Disease

Five Year Old Holly Was Diagnosed With Batten Disease

A New Collaboration Could Bring A New CLN2 Batten Disease Treatment To Patients Faster

LX1004 for CLN2 Batten Disease Earns Rare Pediatric Disease Designation

hCLN5 Gene Therapy for CLN5 Batten Disease Granted EMA Orphan Drug Status

Two Fathers of Daughters with Batten Disease Created a Bill Which Would Fund Genetic Testing

A Batten’s Disease Flair Up Took Their Christmas Spirit. Their Small Town Gave It Back.

Positive Data Announced for CLN6 Batten Disease Gene Therapy AT-GTX-501

Discovering CNL6’s Role in Batten Disease

Free Genetic Testing for Lysosomal Storage Diseases Provided by Invitae, Axovant

Is It Safe to Anesthetize Young Patients with Batten Disease?

It Just Makes “Antisense:” Using Genetic Medicine to Treat Rare Diseases

Faulty Proteins and Failed Genes: Understanding the Biology Behind Congenital Diseases

How Batten Disease has Affected the Carroll Family

Neurogene and Behind the Seizure Work to Provide Better Diagnostics for Batten Disease

Rare Diseases: A Catch 22 Dilemma

A Girl Diagnosed with Batten Disease got Treated with a Drug Developed Specifically for Her

Metastatic Breast Cancer: Navigating Grief

Rethinking What It Means to Live With Acromegaly

The Let’s Chat CAR T One-on-One Mentor Program: Speaking with Someone Who Understands What You Are Going Through

Get Involved

Let’s Work Together!

Keep Up to Date

Learn More