Today, the U.S. Food and Drug Administration approved Miplyffa (arimoclomol), an oral medication for the treatment of Niemann-Pick disease, type C (NPC). Miplyffa, in combination with the enzyme ...

Zevra Therapeutics has won Food and Drug Administration approval of Miplyffa for Niemann-Pick disease type C, making it the first drug cleared in the U.S. for the rare neurodegenerative disorder.

Jenssen guessed then that two of her daughters, the twins, had a Duchenne mutation, and she knew in her heart that, for at ...

The U.S. Food and Drug Administration approved Zevra Therapeutics' drug for a rare and fatal genetic disorder, making it the ...

Sanofi said on Friday that its multiple sclerosis drug candidate was shown to delay worsening of a progressive form of the ...

Immunoglobulin A nephropathy (known as IgA nephropathy or Berger's disease) is a rare kidney disease that occurs when the ...

A House committee on Wednesday passed a bill to renew a program aimed at aiding development of drugs for rare diseases in ...

An NIH-supported clinical trial of pomalidomide in treating hereditary hemorrhagic telangiectasia stopped early due to ...

Passage of the measure would tee it up to be added to government funding legislation. It also would ensure the voucher program would be in place another five years. Meanwhile, the House rejected the ...

A dog with a rare and very serious disease that makes her unable to move her body makes a miraculous recovery after her vet ...

Scientists have made a major breakthrough in medical research with the discovery of a new autoinflammatory disease called Lyn ...

It’s been almost a year since the Food and Drug Administration approved the first genetic treatments for sickle cell disease.