Posts by: admin

admin

Chris Comish serves as the Publisher of the website, and is responsible for directing the editorial focus as well as putting the finishing touches on many featured articles.

April 22, 2020April 22, 2020

TransCon PTH Holds Promise in Treating Hypoparathyroidism, Phase 2 Trial Shows

Hypoparathyroidism News

Ascendis Pharma’s investigational therapy TransCon PTH safely restored calcium and phosphate balance and eliminated the need for current standard care in adults with hypoparathyroidism, top-line data from a Phase 2 ... Read more

April 22, 2020April 22, 2020

Novel IPAH Mutations Found in Chinese Patients, Study Reports 

Pulmonary Hypertension News

Newly identified mutations that contribute to a higher susceptibility of idiopathic pulmonary arterial hypertension (IPAH) in people from China, were found in a pathway commonly targeted by therapies that dilate ... Read more

April 22, 2020April 22, 2020

How Neuropathy Patients Can Socialize During Self-quarantine

FAP News Today

Staying positive and maintaining mental health may be challenging during self-quarantine. For neuropathy patients, whether they have diabetic neuropathy, familial amyloid polyneuropathy, or chronic inflammatory demyelinating polyneuropathy, it may be ... Read more

April 22, 2020April 22, 2020

Here’s How IBS Affects Me

Fibromyalgia News Today

If the statistics are correct, up to 70% of fibromyalgia patients suffer from irritable bowel syndrome (IBS). Few of us need an official awareness month to be reminded of its ... Read more

April 22, 2020April 22, 2020

Giving Thanks for Peace While in Self-quarantine

Hemophilia News Today

If I had to define my self-quarantine in one word, it would be peace. I live in a peaceful, small, rural town. My street is quiet and my porch has a ... Read more

April 22, 2020April 22, 2020

Finding Your Home Base in an Uncertain World

Sanfilippo News

My favorite part of writing columns for Sanfilippo News is the opportunity to reach people both in and outside of the Sanfilippo community. In this quarantine, we’re all sort of ... Read more

April 22, 2020April 22, 2020

The Zeego Tales: A Little Something Extra

Friedreich's Ataxia News

Focusing on times of plenty and excess amid the COVID-19 pandemic seems foreign, or even rude. However, this calamity and all the pain it’s wrought cannot be all we see. ... Read more

April 22, 2020April 22, 2020

Rare Disease Therapy Development: Finding a Way Forward

Batten Disease News

Gaining access to lifesaving or even life-altering treatments has never been easy for many rare disease patients — including those with Batten disease. But the landscape is changing. What felt ... Read more

April 22, 2020April 22, 2020

A Cautionary Tale About Hydroxychloroquine’s Side Effects

Lambert-Eaton News

It seems that one can’t turn on the news, scroll through Twitter, or read Facebook these days without seeing a reference to  the antimalarial drug hydroxychloroquine and what it might ... Read more

April 22, 2020April 22, 2020

Ella Is Finding Ways to Stay Busy During the Quarantine

SMA News Today

Like everybody else, Ella has been kept in the house because of COVID-19. During the school week, she spends much of her morning working on classroom assignments. Each morning she ... Read more

April 21, 2020April 22, 2020

I Wish I Was Sheltered in Place with Anyone but Me

Cystic Fibrosis News Today

I am gravely inflexible. This might seem shocking to those who know me, since I write openly about being a professional dancer. A lot of my career depends on how ... Read more

April 21, 2020April 21, 2020

UCB Acquires Ra Pharma, Adds Zilucoplan to Its MG Pipeline

Myasthenia Gravis News

UCB has completed its acquisition of Ra Pharmaceuticals, priced at about $2.3 billion, meaning that zilucoplan — an investigational treatment for myasthenia gravis (MG) — is being added to UCB’s ... Read more

April 21, 2020April 21, 2020

Bavencio Seen as Ineffective in Phase 3 Head and Neck Cancer Trial

Immuno-Oncology News

A Phase 3 trial of Bavencio (avelumab) in people with squamous cell carcinoma of the head and neck (SCCHN) was ended early due to a lack of effectiveness seen in ... Read more

April 21, 2020April 21, 2020

FDA Places Vutrisiran on Fast Track as Potential FAP Treatment for Adults

FAP News Today

The U.S. Food and Drug Administration (FDA) has granted fast track designation to Alnylam Pharmaceuticals’ vutrisiran as a potential treatment of adults with familial amyloid polyneuropathy (FAP). Fast track is intended to accelerate ... Read more

April 21, 2020April 21, 2020

Stem Cell Transplant Safe, Effective in Halting Disease Progression in Adult-Onset Forms of ALD, Study Shows

Adrenoleukodystrophy News

Stem cell transplant is a safe and effective procedure to halt disease progression in patients with adult-onset forms of adrenoleukodystrophy (ALD), a study says. The study, “Clinical efficacy of haematopoietic ... Read more