The Scientist

» rare diseases

Most Recent

image: Spinoff Company to Develop Rare Disease Drugs

Spinoff Company to Develop Rare Disease Drugs

By | January 7, 2015

Maryland-based Vtesse will work with the US National Institutes of Health to advance treatments for Niemann-Pick Type C disease and other lysosomal storage disorders.

1 Comment

image: Uncommonly Rare

Uncommonly Rare

By | November 1, 2014

How one of the rarest neurodegenerative diseases could lend insight into ubiquitous neuroprotective processes

1 Comment

image: Mosaic Mutations

Mosaic Mutations

By | July 31, 2014

Some genetic abnormalities that appear to have sprung up independently in children are in fact present in a portion of their parents’ cells.

3 Comments

image: NIH Expands Undiagnosed Diseases Program

NIH Expands Undiagnosed Diseases Program

By | July 3, 2014

The National Institutes of Health is funding six extramural medical centers to help identify the causes of mysterious diseases.

0 Comments

image: Gene Therapy Improves Sight

Gene Therapy Improves Sight

By | January 17, 2014

Patients progressing toward blindness now have better vision after a gene therapy trial.

0 Comments

image: Gene Therapy Coming of Age?

Gene Therapy Coming of Age?

By | July 11, 2013

Using lentiviral vectors to replace mutated genes in blood stem cells, scientists successfully treat two rare diseases apparently without causing harmful side effects.

2 Comments

image: Gene Therapy Arrives in Europe

Gene Therapy Arrives in Europe

By | November 6, 2012

The European Commission approves the Western hemisphere’s first gene therapy, aimed at correcting a lipid-processing disorder.

7 Comments

image: Island Disease

Island Disease

By | August 1, 2012

People living on islands in the Norwegian Sea suffer from an unusually high rate of certain genetic diseases and health issues, making the population ripe for research.

3 Comments

image: FDA May Fast-track Needed Drugs

FDA May Fast-track Needed Drugs

By | November 18, 2011

New congressional proposals would allow drug makers to bypass traditional clinical trial requirements for drugs that address rare diseases.

0 Comments

image: One-Eyed Shark

One-Eyed Shark

By | October 19, 2011

This is not an ancient one-eyed species, but an animal suffering from a rare congenital condition known as cyclopia, which has been documented in a variety of species, including humans. 

0 Comments

Advertisement

Popular Now

  1. How Fats Influence the Microbiome
  2. Hearing Help
    Features Hearing Help

    For decades, the only remedies for hearing loss were devices such as hearing aids or cochlear implants. Now, the first pharmaceutical treatments may be on the way.
     

  3. Psychology’s Failure to Replicate
  4. The Great Big Clean-Up
    Features The Great Big Clean-Up

    From tossing out cross-contaminated cell lines to flagging genomic misnomers, a push is on to tidy up biomedical research.

Advertisement
Eppendorf
Eppendorf
Advertisement
Life Technologies