Zevra Therapeutics, Miplyffa and Niemann-Pick Disease
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.
After prior snub, Zevra clinches FDA approval for rare lysosomal storage disorder drug Miplyffa
For Zevra Therapeutics and its rare neurodegenerative disease med arimoclomol, the second time’s the charm. | The FDA approved Zevra’s arimoclomol capsules to treat the rare lysosomal storage disorder Niemann-Pick disease type C in conjunction with Johnson & Johnson’s enzyme inhibitor miglustat.
Zevra Therapeutics' Drug Becomes First FDA-Approved Treatment For Ultra-Rare Neurodegenerative Disease
The FDA has approved Zevra Therapeutics' Miplyffa (arimoclomol) as the first treatment for neurological symptoms of Neimann-Pick disease, type C. This approval follows favorable voting by the Genetic Metabolic Diseases Advisory Committee.
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
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 an orally consumable, allergen-free, food supplement, that has proven to be safe, with the following potentials in DMD:
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.
Legionnaires' disease, Gaston County
New York Post1h
Woman with rare disease claims she’s now blind and bruised after doctors made her get 3 vaccines
A 23-year-old Florida woman has been temporarily blinded and bruised after allegedly being required to receive multiple ...
STAT22h
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 ...
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 ...
MedPage Today19h
First Drug Approved for Niemann-Pick Disease Type C
Opens in a new tab or window The FDA on Friday approved arimoclomol (Miplyffa) as the first treatment specifically for the ...
STAT19h
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 ...
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.
Hosted on MSN7h
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 ...