Month: May 2022

May 31, 2022June 1, 2022

Educational Events on Tap to Mark Dravet Awareness Month and Day

Dravet Syndrome News

From remembering those lost to Dravet syndrome to sharing patient stories, supporters are gearing up to mark Dravet Syndrome Awareness Month, observed each June to call attention to this severe ... Read more

May 31, 2022May 31, 2022

2 Proteins That Silence Fetal Hemoglobin May Be Therapy Target

Sickle Cell Disease News

Interfering with two newly identified proteins — NFIA and NFIX — involved both directly and indirectly in silencing fetal hemoglobin in red blood cells, could be a new therapeutic approach ... Read more

May 31, 2022May 31, 2022

Blood Protein Levels May Help ID Hidden Lung Disease in Sjögren’s

Sjogren's Syndrome News

Elevated levels in the blood of the protein Krebs von den Lungen-6 (KL-6) — an indicator of lung inflammation — may help identify interstitial lung disease (ILD) in people with ... Read more

May 31, 2022May 31, 2022

Urinary Tract Problems May Be Common With IOPD, Despite ERT

Pompe Disease News

Lower urinary tract symptoms, including a loss of bladder control and unusual urine flow, are prevalent among children with classic infantile-onset Pompe disease (IOPD) who begin treatment early with enzyme-replacement therapy ... Read more

May 31, 2022May 31, 2022

What Upstaza’s Likely Approval in Europe Means to Our Community

AADC News

Chimes and notifications began to appear on my phone on the morning of May 20. Messages and questions in my inboxes soon followed. Everyone was excited that the previous day, ... Read more

May 31, 2022May 31, 2022

Resources for Mental Health Are an Important Part of Any Care Plan

COVID-19 Columns, Pulmonary Fibrosis News Columns

Carrying the weight of the world on your shoulders can be exhausting. Those of us touched by rare disease — whether as a patient, caregiver, loved one, or friend — ... Read more

May 31, 2022June 14, 2022

Resources for Mental Health Are an Important Part of Any Care Plan

COVID-19 Columns, Pulmonary Fibrosis News Columns

Carrying the weight of the world on your shoulders can be exhausting. Those of us touched by rare disease — whether as a patient, caregiver, loved one, or friend — ... Read more

May 31, 2022May 31, 2022

DMD Diagnosis Takes as Long Today as Decades Ago, Study Finds

Muscular Dystrophy News

Despite broad initiatives seeking improvements, the time it takes an individual with Duchenne muscular dystrophy (DMD) to get a correct diagnosis has remained largely unchanged over the past three decades, ... Read more

May 31, 2022May 31, 2022

Stem Cells, Turned Into Neurons, Restore Motor Function in Rats

Parkinson's News Today

Stem cells in the lab were turned into nerve cells (neurons) that can make dopamine — the chemical that is lacking in patients with Parkinson’s disease — and transferred into ... Read more

May 31, 2022May 31, 2022

$3.9M Grant Awarded to Study Effects of Low-glycemic Diet

Multiple Sclerosis News Today

The U.S. Department of Defense (DOD) has awarded $3.9 million to fund clinical research to test the impact of a low-glycemic diet on physical, cognitive, and psychological function in people ... Read more

May 31, 2022May 31, 2022

Connecticut Agrees to Establish Its Own Rare Disease Advisory Council

Lambert-Eaton News

Lawmakers in Connecticut agreed to set up a permanent Rare Disease Advisory Council (RDAC), an advisory body working to better address the unique needs of people with rare diseases like Lambert-Eaton ... Read more

May 31, 2022May 31, 2022

Activities Mark Alzheimer’s and Brain Awareness Month Across June

Alzheimer's News Today, COVID-19 News

The start of summer holds the promise of beach trips, barbecues, and vacations, but for people with Alzheimer’s disease and their caregivers, family, and friends, June also means Alzheimer’s and Brain ... Read more

May 31, 2022May 31, 2022

Mayzent and Kesimpta Gaining Ground as MS Treatments in Canada

Multiple Sclerosis News Today

Novartis’ Mayzent (siponimod) and Kesimpta (ofatumumab) are gaining ground among multiple sclerosis (MS) therapies in Canada, according to the latest Spherix Global Insights’ report. “Following an eventful 2021 that included the ... Read more

May 31, 2022May 31, 2022

$2.7M NIH Grant Supports Work Into Stem Cell Role in Lung Inflammation

Cystic Fibrosis News Today

A $2.7 million grant from the National Heart, Lung, and Blood Institute, part of the National Institutes of Health (NIH), has been awarded to two stem cell researchers at the ... Read more

May 31, 2022May 31, 2022

Masitinib Being Reviewed for OK in Canada as Add-on ALS Therapy

ALS News Today

Health Canada has started to review AB Science’s application requesting that masitinib be approved as an add-on therapy for amyotrophic lateral sclerosis (ALS). The application will be reviewed under the notice ... Read more