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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.
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.
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.
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 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).
STAT
17h
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 ...
FierceBiotech
2d
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 ...
Bored Panda on MSN
1d
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 ...
STAT
21h
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 ...
Targeted Oncology
1d
Atypical Chronic Myeloid Leukemia: A Rare Disease With Poor Prognosis
Ongoing research is exploring new molecular targets and targeted therapies for this challenging disease.
Psychology Today
1d
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 ...
2d
on 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 ...
ktvq
12h
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 ...
17m
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 ...
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