Latest News

Friday, January 19, 2018
This RSS feed URL is deprecated, please update. New URLs can be found in the footers at https://news.google.com/news
Friday, January 19, 2018

UW Today

University Faculty Lecture to highlight screening newborns for genetic diseases
UW Today
But sometimes, due to random genetic mutation, these enzymes struggle to perform their proper roles, causing rare and often fatal diseases in babies and children. Gelb and his colleagues have worked to develop screens that can accurately test in babies ...

and more »
Friday, January 19, 2018

Rare Disease Report

Orphazyma A/S Granted Rare Pediatric Disease Designation for NPC Treatment
Rare Disease Report
Arimoclomol, in development by Danish biotech Orphazyme A/S for the treatment of Niemann-Pick Disease Type C (NPC), was granted rare pediatric disease designation by the U.S. Food and Drug Administration (FDA) this morning, the company announced ...

and more »
Friday, January 19, 2018

A Rundown Of Dravet Syndrome Drugs In The Pipeline
Nasdaq
(RTTNews.com) - Rare commodities are worth more than good is a Chinese adage. And more so when it is in the field of medicine. Rare diseases, also called orphan diseases, as the name implies, affect very small numbers of patients. Estimates by the ...

and more »
Friday, January 19, 2018

Coherent News (press release) (blog)

Global Rare Disease Treatment Market is Projected to Grow US$ 78434 Mn by the end of 2025
Industry Today (press release)
A comprehensive evaluation presented by Persistence Market Research on the global rare disease treatment marketin a new research report titled “Rare Disease Treatment Market: Global Industry Analysis 2012 – 2016 and Forecast 2017 – 2025” highlights the ...
Global Rare Disease Drugs Market – Competitive AnalysisCoherent News (press release) (blog)
Global Rare Disease Drug Market : Top Manufacturers, Market Growth Analysis, Sales, Revenue, Gross Margin ...Carelton Free Press
Global Rare Disease Drug Market 2018- Pfizer and Inc, Eli Lilly and Company, Novo Nordisk A/S, AstraZenecaTimeGazette
Herald Keeper
all 80 news articles »
Friday, January 19, 2018

Arimoclomol for NPC receives Rare Pediatric Disease Designation
GlobeNewswire (press release)
Copenhagen, Denmark, January 19, 2018 – Orphazyme A/S, a Danish biotech company listed on Nasdaq Copenhagen (TICKER: ORPHA.CO), with a late-stage, orphan-drug pipeline, today announced that arimoclomol has been granted rare pediatric disease ...

and more »
Thursday, January 18, 2018

The Sun

Woman, 20, is trapped in the body of a girl HALF her age by rare disease – but dreams of becoming a doctor
The Sun
She also has a number of the secondary characteristics, such as dwarfism, cardiomyopathy (a type of heart disease), chronic pulmonary lung disease, short limbs, fragile bones and alopecia. But she refuses to be held back by her condition, Mary said ...

Thursday, January 18, 2018

Patients with rare disease appeal Karnataka govt to release funds
United News of India
Patients with rare disease appeal Karnataka govt to release funds. Bengaluru, Jan 18 (UNI) People sufferring from rare disease have appealed to the Karnataka Government to release funds for treatment. The State Government had initially provided a grant ...

and more »
Thursday, January 18, 2018

Otaku Reports

Rare Disease Drug Market Usage, Dosage And Side Effects Analysis 2018
Otaku Reports
Global Rare Disease Drug Market Research Report 2018 presents an in-depth assessment of the Rare Disease Drug including enabling technologies, key trends, market drivers, challenges, standardization, regulatory landscape, deployment models, operator ...
Global Rare Disease Drug Market 2018 – Merck & Co., Inc., Johnson & Johnson, Biogen, Inc., DecipheraThe Financial Portal
Global Rare Disease Drug Market Attractiveness, Competitive Landscape and Key Players Novartis AG , Bristol-Myers ...Island Post Gazette

all 18 news articles »
Wednesday, January 17, 2018

Daily Mail

You'd never know! Little boy who lost his eye to rare disease after his mother spotted a golden 'glow' in a baby ...
Daily Mail
In 2015, New Zealand mother Sarah Bowers, 31, spotted a faint golden glow in her then 16-week-old baby boy Mason's eye after taking photos of him playing in the bath tub. What followed was a series of terrifying specialist appointments and an operation ...

and more »