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.

0 Comments

image: Uncommonly Rare

Uncommonly Rare

By | November 1, 2014

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

0 Comments

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. Antibiotics and the Gut Microbiome
  2. Sex Differences in Pain Pathway
  3. The Sum of Our Parts
    Features The Sum of Our Parts

    Putting the microbiome front and center in health care, in preventive strategies, and in health-risk assessments could stem the epidemic of noncommunicable diseases.

  4. Anti-Vax Doctor Found Dead
    The Nutshell Anti-Vax Doctor Found Dead

    Police are calling the death of James Bradstreet, a physician who claimed vaccines cause autism and offered autism cures to patients, an apparent suicide.

Advertisement
Bina Technologies
Bina Technologies
Advertisement
The Scientist