Zevra Therapeutics, Miplyffa and Niemann-Pick Disease
US FDA approves Zevra's treatment for rare genetic disease
The U.S. Food and Drug Administration has 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.
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 that a planned advisory committee meeting won’t be required. | After pushing back the decision date for Applied Therapeutics’ metabolic disorder drug govorestat,
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
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.
FDA pushed to be more open, collaborative to accelerate rare disease R&D
The FDA should be more open and collaborative to unleash a surge in approvals of rare disease drugs, according to a report by the National Academies of Sciences, Engineering, and Medicine.
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.
Legionnaires' disease, Gaston County
New York Post3h
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 ...
Commentary: Congress has just weeks to help children with rare diseases
A rare-disease federal incentive program critical to biotech R&D is in jeopardy, writes Leslie Williams, president and CEO of ...
STAT1d
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 ...
Psychology Today2d
How I Survived a Rare Autoimmune Disease
I walked out of my marriage and immediately fell ill. I was ill for two years. At first, I thought it was a cold that would ...
STAT21h
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 Oncology2d
Atypical Chronic Myeloid Leukemia: A Rare Disease With Poor Prognosis
Ongoing research is exploring new molecular targets and targeted therapies for this challenging disease.
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Calves to Cure helps fund research for Worden boy's rare disease
The Billings Livestock Commission hosted the sixth annual Calves to Cure on Thursday, an event to help raise money and ...
Business Wire13h
Neu-REFIX Beta glucan receives rare pediatric disease and orphan drug designations from the US FDA for treatment of Duchenne Muscular Dystrophy
Neu refix granted orphan disease designation (ODD) & rare pediatric disease designation (RPDD) by US FDA for treatment of ...