Month: November 2021

November 30, 2021December 1, 2021

Lung Function Reduced in LOPD Despite Long-term Myozyme Use

Pompe Disease News

Despite treatment for years with Myozyme (alglucosidase alfa) — marketed in the U.S. as Lumizyme — people with late-onset Pompe disease (LOPD) still develop airway abnormalities and experience reduced lung function, ... Read more

November 30, 2021November 30, 2021

LEMS in Lung Cancer Patient Given Tecentriq Shows Need for Vigilance

Lambert-Eaton News

The rare case of a man with advanced small cell lung cancer (SCLC) who went on to develop Lambert-Eaton myasthenic syndrome (LEMS) after receiving treatment with Tecentriq (atezolizumab), an immune ... Read more

November 30, 2021November 30, 2021

Trial Data Show STK-001 Safe, Likely Effective at Reducing Seizures

Dravet Syndrome News

Stoke Therapeutics’ investigational therapy STK-001 is generally safe and results in a trend toward a reduction in seizure frequency in children and adolescents with Dravet syndrome, according to interim data ... Read more

November 30, 2021November 30, 2021

2 Blood Biomarkers May Be Used to Monitor Lecanemab’s Efficacy

Alzheimer's News Today

The blood levels of two biomarkers of Alzheimer’s disease are significantly associated with changes in brain beta-amyloid deposits and cognitive function in early Alzheimer’s patients treated with lecanemab (BAN2401), according ... Read more

November 30, 2021November 30, 2021

How Pivoting My Perspective Helps Me Live With ALS

ALS News Today Columns

I spent most of my life following daily habits and routines that shaped my perspective about how life ought to be. When ALS appeared, it brought change and created imaginary ... Read more

November 30, 2021November 30, 2021

Oral Oxbryta Safe, Effective Over Long Term and in Real World

Sickle Cell Disease News

Long-term treatment with Oxbryta (voxelotor) safely and effectively reduced anemia and red blood cell destruction (hemolysis), while keeping a low rate of painful vaso-occlusive crises (VOCs), in adolescents and adults ... Read more

November 30, 2021November 30, 2021

Investing in Home Healthcare Will Support Patients, Caregivers

COVID-19 Columns, Pulmonary Hypertension News Columns

The tightness creeps up in my lungs just as I’m about to make up the bed with a second clean sheet. I pause to catch my breath and wonder how ... Read more

November 30, 2021November 30, 2021

Rates of SSc, Raynaud’s Likely Higher Among Post-9/11 Veterans

Scleroderma News

A higher rate of systemic sclerosis (SSc), very early diagnosis of systemic sclerosis (VEDOSS), and Raynaud’s phenomenon is evident among U.S. military veterans deployed in support of post-9/11 operations, compared ... Read more

November 30, 2021November 30, 2021

US Trial of Gene Therapy AMT-130 Enrolling High-dose Patient Group

Huntington's Disease News

A Phase 1/2 clinical trial testing uniQure’s potential gene therapy AMT-130 in people with Huntington’s disease can continue enrollment, according to a positive review by the study’s independent data safety monitoring board ... Read more

November 30, 2021November 30, 2021

Rare Case Report Attributes MG in Elderly Man to COVID-19 Vaccine

COVID-19 News, Myasthenia Gravis News

An 82-year-old man was diagnosed with myasthenia gravis (MG) shortly after receiving his second dose of the BioNTech/Pfizer COVID-19 vaccine, a case study reports. He lacked typical MG symptoms, but ... Read more

November 30, 2021November 30, 2021

Obesity May Cause ‘Subtle’ Heart Changes Starting in Early Adulthood

Prader-Willi News

Subtle heart changes in people with Prader–Willi syndrome (PWS) may begin relatively early in adulthood, and being overweight may be related to these cardiovascular abnormalities, a small study reported. “We ... Read more

November 30, 2021November 30, 2021

Lung and Nutritional Health, Like CF Mutation, Can Affect Lifespan

Cystic Fibrosis News Today

Inadequate nutrition, poorly working or chronically infected lungs, and mutations leading to more severe disease can shorten the lifespan of people with cystic fibrosis (CF), a study in adult patients ... Read more

November 30, 2021November 30, 2021

Some Cell Therapies Work by Dying, New Research Shows

Multiple Sclerosis News Today

Certain cell therapies that hold promise for treating autoimmune diseases like multiple sclerosis might work not because of the way the cells live in the body during treatment, but because ... Read more

November 30, 2021November 30, 2021

Phase 3 Trial of Masitinib in Slowing Progression Again Enrolling in US

ALS News Today

The U.S. Food and Drug Administration (FDA) has cleared AB Science to resume patient enrollment in its confirmatory Phase 3 trial investigating masitinib as an add-on treatment in people with amyotrophic ... Read more

November 30, 2021November 30, 2021

PF Warriors, Joe & Bella Team Up to Help Patients in Holiday Shopping

COVID-19 News, Pulmonary Fibrosis News

PF Warriors has partnered with online shopping site Joe & Bella to make holiday gift-giving easier for people with pulmonary fibrosis (PF) — while raising funds for the nonprofit’s mission ... Read more