Rare Disease Report


In a story written by Jackie MacMullan and published on ESPN.com, it was revealed the Hall of Fame basketball player Nate “Tiny” Archibald has been living with amyloidosis.
Alnylam has announced this afternoon that the U.S. FDA has accepted for filing its New Drug Application for patisiran, an investigational RNAi therapeutic targeting transthyretin for the treatment of hATTR amyloidosis.
Alnylam announced that its Marketing Authorisation Application for patisiran was accepted by the European Medicines Agency.
Today, Eiger BioPharmaceuticals announced that results from the Phase 2 LIBERTY study demonstrated no improvement overall in its Phase 2 PAH treatment study, evaluating the effects of ubenimex.
Filmmaker Kumail Nanjiani opened up on Twitter – perhaps more candidly than ever before – after his wife's mother found an old hospital badge.
Biopharmaceutical company AbbVie has announced the start of the 2017 Thriving Student Scholarship contest for cystic fibrosis patients.
Like many lipodystrophy patients, she appeared to be very healthy. But the disease is a silent killer.
There are currently 75 Progeria children in clinical trials but with 15 compounds under consideration for trials, the math doesn't work.
At WORLDSymposium, Broomfield et al performed a retrospective review of all Hunter syndrome [mucopolysaccharidosis (MPS) II] patients managed at Royal Manchester Children's Hospital, Manchester, United Kingdom since the commercial introduction of Elaprase (idursulfase).
A recently published review article in Expert Opinion of Orphan Drugs, Drs Spinazzola and Kunkel provide an overview of the leading drugs in development for treating the secondary effects of DMD.
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