rare disease news - Search News

US FDA approves Zevra's treatment for rare genetic disease

The U.S. Food and Drug Administration approved Zevra Therapeutics' drug for a rare and fatal genetic disorder, making it the first treatment to get a nod for the condition, the health regulator said on Friday.

YAHOO!Finance · 25m

Zevra Therapeutics' Drug Becomes First FDA-Approved Treatment For Ultra-Rare Neurodegenerative Disease

Miplyffa (arimoclomol), an oral medication for Neimann-Pick disease, type C (NPC). Miplyffa, in combination with Johnson & Johnson's (NYSE:JNJ) enzyme inhibitor Zavesca (miglustat), is approved to treat neurological symptoms associated with NPC in adults and children two years of age and older.

Business Insider · 41m

Zevra Therapeutics’ MIPLYFFA™ (arimoclomol) Receives U.S. FDA Approval as Treatment for Niemann-Pick Disease Type C

MIPLYFFA is the first FDA-approved treatment for Niemann-Pick disease type C (NPC), an ultra-rare and progressive

MarketWatch · 1h

FDA Approves Zevra's Miplyffa for Niemann-Pick Disease Type C -- 2nd Update

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

Daily Journal · 2h

FDA Approves First Treatment for Niemann-Pick Disease, Type C

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

FDA approves first drug for rare neuron disease, Niemann-Pick type C

The Food and Drug Administration on Friday approved the first treatment for Niemann-Pick disease, type C, the culmination ...

Bored Panda on MSN1d

Dog Has A Rare Disease That Makes Her Body Frozen, Surprises Everyone And Recovers Against All Odds

For example, this one vet chose to care for this pitbull puppy despite the fact that she had a rare and very serious disease ...

FierceBiotech2d

FDA scraps adcomm for Applied's rare disease drug ahead of November decision

After pushing back the decision date for Applied Therapeutics’ metabolic disorder drug govorestat, the FDA has now decided ...

House panel passes pediatric rare disease bill, setting it up for government funding bill

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

19m

Neu-REFIX Beta Glucan Receives Rare Pediatric Disease And Orphan Drug Designations From The US FDA For Treatment Of Duch...

The Neu-REFIX Beta 1,3-1,6 glucan, is an exo-polysaccharide produced by N-163 strain of Aureobasidium Pullulans, available as ...

Targeted Oncology1d

Atypical Chronic Myeloid Leukemia: A Rare Disease With Poor Prognosis

Ongoing research is exploring new molecular targets and targeted therapies for this challenging disease.

2don MSN

Health officials in L.A. Co. investigating rare disease spread from raccoons to humans

The Los Angeles County Department of Public Health is looking into two human cases of a rare parasitic infection that comes ...

Hosted on MSN5h

How To Treat IgA Nephropathy (Berger's Disease)

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

Results that may be inaccessible to you are currently showing.

Hide inaccessible results