Month: April 2022

April 29, 2022April 29, 2022

Within 3 Years, This Column Has Grown and Changed

Somehow, it’s already been three years since I began sharing my story with you in this column. It’s been a beautiful, heartfelt adventure that I’ve thoroughly enjoyed. But I have to ... Read more

April 29, 2022April 29, 2022

The MSer Who Lay in Bed for 2 Years and Can Now Walk Again

Multiple Sclerosis News Today Columns

My column’s handle is “Fall Down, Get Up Again” because the first piece I wrote for Multiple Sclerosis News Today was titled “A Mountain to Climb with MS — in ... Read more

April 29, 2022April 30, 2022

Adrenal Removal Surgery Linked to Better Survival in Ectopic Cushing’s

Cushing's Disease News

Undergoing a surgical removal of the adrenal glands — called adrenalectomy — was associated with a better prognosis than cortisol-suppressing therapies alone in people with ectopic Cushing’s syndrome (ECS) caused ... Read more

April 29, 2022April 30, 2022

#AAN2022 – Allopregnanolone Found Safe, May Ease Brain Atrophy

Alzheimer's News Today

The therapeutic candidate allopregnanolone is safe and well-tolerated in people with early Alzheimer’s disease, according to data from a Phase 1b/2a clinical trial that also investigated the medication’s maximum-tolerated dose. ... Read more

April 29, 2022April 29, 2022

Online Survey to Assess Stigma’s Impact in ALS Patients, Caregivers

ALS News Today

An international team of researchers is asking adults with amyotrophic lateral sclerosis (ALS) and their family caregivers to complete an online survey about their experiences with stigma around the disease. ... Read more

April 29, 2022April 29, 2022

Organizations Rally to Help Ukrainian Rare Disease Patients

Ehlers-Danlos News

A Russian military plane crash near Tetiana Zamorska’s home in Kyiv, Ukraine, was a sign that it was time for her and her family to leave. The treacherous, 34-hour pilgrimage that ... Read more

April 29, 2022April 29, 2022

Organizations Rally to Help Ukrainian Rare Disease Patients

Hypoparathyroidism News

A Russian military plane crash near Tetiana Zamorska’s home in Kyiv, Ukraine, was a sign that it was time for her and her family to leave. The treacherous, 34-hour pilgrimage that ... Read more

April 29, 2022April 29, 2022

Vicore Trial Testing New AI App for Anxiety in IPF Enrolls 1st Patient

Pulmonary Fibrosis News

The first patient has been enrolled in a U.S. clinical trial evaluating the efficacy and safety of a new artificial intelligence-based app, called Almee, that’s designed to treat anxiety in ... Read more

April 29, 2022April 30, 2022

PoNS Device for MS-associated Gait Problems Now Available in US

Multiple Sclerosis News Today

The portable neuromodulation stimulator (PoNS) device, which improves walking abilities in people with multiple sclerosis (MS), is now available commercially in the U.S. for patients ages 22 and older who ... Read more

April 29, 2022April 29, 2022

HDAC6 Inhibitors Restore Neuromuscular Defects in Zebrafish

Charcot-Marie-Tooth News

Treatment with inhibitors of the HDAC6 protein, including the new compound CKD-504, was able to restore proper connections between nerves and muscles and improve movement in zebrafish with low levels of ... Read more

April 29, 2022April 29, 2022

PRX-102 Shows Safety, Efficacy Similar to Fabrazyme in Phase 3 Trial

Fabry Disease News

Two years of treatment with the experimental enzyme replacement therapy PRX-102 (pegunigalsidase alfa) prevented kidney decline in adults with Fabry disease and showed safety and effectiveness similar to Fabrazyme (agalsidase beta), ... Read more

April 29, 2022April 29, 2022

Immune NKT Cells May Suggest Early Heart Involvement

Sarcoidosis News

The blood levels of natural killer T-cells (NKT), a type of immune cell, are reduced significantly in sarcoidosis patients with subclinical signs of heart involvement relative to those without, a ... Read more

April 29, 2022April 29, 2022

Protecting My Health Against Stress Is Essential

COVID-19 Columns, Cystic Fibrosis News Today Columns

The world demands my constant attention. Everything that comes across my news feed is presented as an emergency. The pressure to know the latest and greatest is at an all-time ... Read more

April 29, 2022April 29, 2022

Why Pool Therapy Is Part of Our Care Plan

Muscular Dystrophy News Columns

I write my columns on Tuesdays. I do this because I procrastinate. My deadline is Wednesday morning, and I’m not about to wake up early on Wednesdays to get it ... Read more

April 29, 2022April 29, 2022

For the Immunosuppressed, Life Still Isn’t Back to ‘Normal’

COVID-19 Columns, Hemophilia News Today Columns

In addition to having hemophilia B and von Willebrand disease, I also am a caregiver to my 79-year-old mother, whom I live with in Florida. Avoiding COVID-19 has been difficult ... Read more