Cyclo Therapeutics’ Trappsol Cyclo was able to induce clinical improvement in seven out of eight children with Neimann-Pick ...
In the space of four days, the number of FDA-approved therapies for ultra-rare genetic disorder Niemann-Pick disease has gone from zero to two, transforming the prospects for patients. IntraBio's ...
Zevra Therapeutics' Miplyffa has become the first medicine to be approved by the FDA for the ultra-rare genetic disorder Niemann-Pick disease. The US regulator has cleared Miplyffa (arimoclomol ...
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2 ‘Strong Buy’ Growth Stocks Poised for 141% to 193% Gains, According to Wall StreetThe biotechnology sector is filled with high-potential growth stocks. With advancements in artificial intelligence (AI), biotech remains at the cutting edge of medical and scientific innovation, ...
What is Niemann-Pick disease type C? Niemann-Pick disease type C is a rare neurodegenerative disorder caused by cholesterol transport issues, leading to severe neurological impairments.
To report SUSPECTED ADVERSE REACTIONS, contact Zevra Therapeutics, Inc. at toll-free phone 1-844-600-2237 or FDA at 1‐800-FDA-1088 or .
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