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Healthline11mon
Overview of Niemann-Pick Disease Type C
But it can occur in people of any ethnic background. Signs and symptoms of Niemann-Pick type C disease depend on your age of onset. Symptoms related to problems with your internal organs tend to ...
New FDA-approved drug offers hope for Niemann-Pick Type C patients
Niemann-Pick Type C, or NPC is a relentlessly progressive and fatal genetic disorder that impacts both the body and the brain ...
pharmaphorum8mon
FDA clears first drug for Niemann-Pick disease
Zevra Therapeutics' Miplyffa has become the first medicine to be approved by the FDA for the ultra-rare genetic disorder Niemann-Pick disease ... to treat neurological symptoms in adults and ...
Nasdaq6mon
Zevra Launches Niemann-Pick Disease Type C Drug Miplyffa In The U.S.
Miplyffa, in combination with the enzyme inhibitor miglustat, was approved by the FDA in September of this year to treat neurological symptoms associated with Niemann-Pick disease type C (NPC ...
MedCity News8mon
FDA Approval Makes a Zevra Drug the First Therapy for Rare and Fatal Metabolic Disease
The drug, acrimoclomol, treats Niemann-Pick disease type C (NPC ... The higher the score, the more severe the disease symptoms. Patient scores in the study showed that Miplyffa led to slower ...
The American Journal of Managed Care2mon
5 Rare Diseases That Now Have Their First FDA-Approved Treatments
Niemann-Pick Disease, Type C: Arimoclomol (Miplyffa ... for an average of 13 years and experience progressive neurological symptoms. In combination with miglustat, arimoclomol aims to mitigate ...
Austin American-Statesman7mon
Austin's IntraBio gets FDA approval for Niemann-Pick Type C1 treatment. What to know.
Abby, then 2, had no symptoms but was diagnosed early because of her sister's diagnosis. The life expectancy for a child with Niemann-Pick is 13 years because of the damage the disease does to the ...
Healio8mon
FDA approves second Niemann-Pick disease type C treatment
Please provide your email address to receive an email when new articles are posted on . The FDA has approved Aqneursa as an oral treatment for neurological symptoms associated with Niemann-Pick ...
FiercePharma8mon
After landmark rare disease approval, IntraBio ready to 'hit the ground running' with Aqneursa launch
as the second therapy to treat neurological symptoms of the lysosomal storage disorder Niemann-Pick disease type C (NPC). IntraBio’s green light came right after Zevra Therapeutics scored a ...
FiercePharma8mon
IntraBio muscles in on Zevra's turf after companies' back-to-back lysosomal storage disorder nods
While it was just last week that Zevra Therapeutics’ Miplyffa became the first FDA-approved treatment for the lysosomal storage disorder Niemann-Pick ... signs and symptoms of the disease ...
Alzheimer's Society3mon
Niemann-Pick disease type C
Niemann-Pick disease type C is one of a group of rare inherited disorders ... loss of movement and difficulties with walking and swallowing. People who first show symptoms in late adolescence or early ...
Medical Xpress7mon
FDA approves Miplyffa for treatment of Niemann-Pick disease, type C
The U.S. Food and Drug Administration has approved Zevra Therapeutics' Miplyffa (arimoclomol), an oral medication for the treatment of Niemann-Pick disease, type C (NPC). Miplyffa, in combination ...