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(Reuters) -Amicus Therapeutics' treatment for a rare muscle disorder called Pompe disease was approved by the U.S. health regulator, the drugmaker said on Thursday, ending its years-long efforts ...
In 2016 Amicus acquired MiaMed which was working on drugs to treat CDKL5 deficiency; Amicus paid $1.8M in cash and around $4.7M in stock, with $83M in biodollars. [ 18 ] After Scott Gottlieb became FDA commissioner in 2017, the CEO of Amicus began lobbying him directly for the FDA to accept the NDA and in February 2018 the FDA accepted it and ...
In 2004, he became a director at Amicus Therapeutics, based in Cranbury, New Jersey, and in January 2005, he was named the president and CEO of the company. [17] Amicus works to develop treatments for rare, devastating genetic disorders. The company has a Pompe treatment in development, as well as Galafold , which is for Fabry disease.
Morgan Stanley downgraded Amicus Therapeutics Inc (NASDAQ:FOLD). Over the last year, the company has executed well commercially, met its goals financially by achieving nonGAAP profitability for ...
Spinal Cord Treatment Restores Function For Paralyzed Patients In Study: ‘New Hope’ ... Spinal cord stimulation restores movement for people with 'muscle wasting' disorder. Show comments ...
Distal muscular dystrophy, also called distal myopathy, is essentially any muscle disease that preferentially affects the hands and/or feet, a much less common pattern than proximal muscle weakness. Late adult-onset type 1; Late adult-onset type 2a; Late adult-onset type 2b; Early adult-onset type 1; Early adult-onset type 2; Early adult-onset ...
Inclusion body myopathy with early-onset Paget disease and frontotemporal dementia (IBMPFD), now more commonly referred to as multisystem proteinopathy (MSP), is an autosomal dominant condition caused by mutations in VCP, HNRPA2B1 or HNRNPA1; it is a multisystem degenerative disorder that can affect muscle, bone, and/or the central nervous system.
The U.S. FDA has approved privately held Italfarmaco Group's drug to treat Duchenne muscular dystrophy (DMD), an inherited muscle-wasting disorder, the health regulator said on Thursday. The oral ...
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