Posts by: Marisa Wexler MS

Marisa Wexler MS

August 4, 2022August 4, 2022

Children With CLN2 Show Eye Degeneration Despite Treatment

Batten Disease News Columns, COVID-19 Columns

Children with late infantile Batten disease on approved enzyme replacement therapy show signs of age-related degeneration in the light-sensing part of the eyes, a new study shows. The study, “Ongoing retinal ... Read more

August 4, 2022August 4, 2022

US Study Finds Delays in CF Care for Babies From Minority Groups

Cystic Fibrosis News Today

Among babies with cystic fibrosis (CF) identified via newborn screening, there are marked delays in the start of care for children from racial and ethnic backgrounds that have historically been ... Read more

August 2, 2022August 2, 2022

Phase 1 Trial of KB407, Gene Therapy for All With CF, to Open in US

Cystic Fibrosis News Today

A Phase 1 clinical trial of an experimental inhalation gene therapy for cystic fibrosis (CF) — KB407 by Krystal Biotech — was given clearance to start by the U.S. Food and Drug ... Read more

July 28, 2022July 28, 2022

CBD Fails to Reduce Seizures in Infantile Batten Mouse Model

Batten Disease News Columns

Six months of treatment with cannabidiol (CBD) reduced brain inflammation in a mouse model of infantile Batten disease, but did not affect the frequency of seizures or the survival of ... Read more

July 27, 2022July 27, 2022

Scientists Test New Method That Can Help Diagnose AADC Deficiency

AADC News

Scientists have discovered a new technique to measure the activity of the aromatic-L-amino acid decarboxylase (AADC) enzyme to help diagnose AADC deficiency. The assay, which uses liquid chromatography-tandem mass spectrometry, ... Read more

July 27, 2022July 27, 2022

Novel Machine Learning Technique May Help ID Tremor Specific to MS

Multiple Sclerosis News Today

A new machine learning strategy was able to differentiate tremor specific to multiple sclerosis (MS) from tremor related to other diseases, with more than 90% accuracy, according to a new ... Read more

July 26, 2022July 26, 2022

Review Gives ‘Cautious Support’ to Childhood Trauma as MS Risk Factor

Multiple Sclerosis News Today

Most available studies suggest a connection between childhood trauma and multiple sclerosis (MS), from earlier symptom onset to potentially poorer outcomes, but more research is needed to understand this association, a review ... Read more

July 25, 2022July 25, 2022

Digoxin, Heart Medication, Helps With Remyelination in Early Study

Multiple Sclerosis News Today

Digoxin, an approved medication for certain heart conditions, promoted the repair of myelin — the protective sheath around nerve fibers that is progressively lost in multiple sclerosis — in mouse models ... Read more

July 25, 2022July 25, 2022

GLP-1 Hormone Infusions May Boost Insulin Production in CF Patients

Cystic Fibrosis News Today

Infusions with a hormone called GLP-1 boosted insulin production in cystic fibrosis (CF) patients with impaired glucose tolerance, according to data from a small study. The results suggest that GLP-1 ... Read more

July 22, 2022July 22, 2022

Inhaled Antibiotics Tied to Greater Risk of Aspergillus Infection

Cystic Fibrosis News Today

People with cystic fibrosis (CF) who use inhaled antibiotics are at more than three times higher risk of becoming infected with the fungus Aspergillus fumigatus, according to a new study. Study ... Read more

July 21, 2022July 21, 2022

Psych Meds May Cause Hyperthermia in Juvenile Batten Patients

Batten Disease News Columns

People with juvenile Batten disease may be at increased risk of developing an abnormally high body temperature when taking certain medications commonly prescribed to manage mental health issues, according to ... Read more

July 21, 2022July 21, 2022

Semi-synthetic Compound Promotes Myelin Repair in MS Mice

Multiple Sclerosis News Today

A man-made molecule derived from a naturally occurring amino acid was able to promote the repair of the myelin sheath — a fatty coating around nerve fibers that is damaged in ... Read more

July 21, 2022July 22, 2022

Abortion Bans Are Likely to Restrict Care for MS Patients: Study

Multiple Sclerosis News Today

Bans on abortion and other reproductive care limitations are likely to have a profound impact on the care of people with multiple sclerosis (MS) and other neurological conditions following the ... Read more

July 20, 2022July 20, 2022

Gene Therapy Upstaza OK’d in Europe to Treat AADC Deficiency

AADC News

The European Commission has approved the gene therapy Upstaza (eladocagene exuparvovec) to treat aromatic l-amino acid decarboxylase (AADC) deficiency in patients ages 18 months and older. The approval covers all ... Read more

July 19, 2022July 19, 2022

Natalizumab, Tysabri Biosimilar, Up for Approval in Europe

Multiple Sclerosis News Today

The European Medicines Agency (EMA) has agreed to review a request to approve Polpharma Biologics‘ biosimilar natalizumab for the treatment of multiple sclerosis (MS). This marks the first time the regulatory ... Read more