10 companies driving forward the rare disease market
Authored by Liftstream
Posted in Orphan Drugs and Rare Diseases
Tagged Aegerion, Amicus Therapeutics, Duchenne Muscular Dystrophy, Liftstream Rare Disease Day, Oxyrane, Promethera, Prosensa, PTC Therapeutics, Rare Disease, Rare Disease Day 2013, Sangart, Sarepta Therapeutics, Synageva, Ultragenyx, White Paper, world orphan drug congress
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PTC Therapeutics opts for conditional approach in Duchenne
Authored by Paul Foster
Posted in Orphan Drugs and Rare Diseases
Tagged Allan Jacobson, AstraZeneca, Ataluren, celgene, Cystic Fibrosis, DMD, DNA, Duchenne Muscular Dystrophy, EMA, FDA, Genzyme, Gilead, Liftstream Rare Disease Day, MAA, Marketing Authorisation Application, Merck, Orphan Drug, Pfizer, PTC Therapeutics, Rare Disease, rare disease day, RDD13, Stuart W. Peltz
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Prosensa & GSK pool resources to drive Drisapersen in Duchenne
Authored by Karl Simpson
Posted in Orphan Drugs and Rare Diseases
Tagged Abingworth, David Mott, DMD, Drisapersen, Duchenne, Dystrophin, Exon 51, Genzyme, GSK, GSK’s Centre of Excellence for External Drug Discovery, Hans Schikan, Henri Termeer, Huntington's, Leiden University Medical Center, Massachusetts, MedImmune, mRNA, New Enterprise Associates (NEA), Phase II, PRO051, Prosensa, The New England Journal of Medicine
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Sarepta’s hopes high for first Duchenne (DMD) therapy
Authored by James Sheppard
Posted in Orphan Drugs and Rare Diseases
Tagged AVI Biopharma, AVI-7100, Department of Defense, DMD, Duchenne Muscular Dystrophy, Ebola, EMEA, Eteplirsen, exon skipping, FDA, Infectious Disease, Influenza, Liftstream Rare Disease Day, Marburg Virus, NIH Clinical Center in Bethesda, Phase II Trials, PMO Chemistry, Rare Disease Day 2013, RDD13, Sarepta Therapeutics, UCL, University College London, USA
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Synageva offering LSD patients renewed hope
Authored by James Sheppard
Funding successes allow Promethera to look to clinical data in 2013
Authored by James Sheppard
Posted in Orphan Drugs and Rare Diseases
Tagged Allogenic Liver Cells, Boehringer Ingelheim, clinical, Crigler-Najjar, Eric Halioua, Etienne Sokal, funding, GMP, HepStem, Liftstream, Liftstream Rare Disease Day, Liver Fibrosis, Phenylketonuria, Promethera Bioscience, rare disease day, RDD13, regenerative medicine, Shire, Stem Cells
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Ultragenyx focused on ultra-rare disease future
Authored by Paul Foster
Posted in Orphan Drugs and Rare Diseases
Tagged 2013, Baylor Research Institute, BioMarin, biotech, Emil Kakkis, Fatty Acid Oxidisation Disorders, funding, Genzyme, Heriditary Inclusion Body Myopathy, IPO, Liftstream, Rare Disease, rare disease day, Sanofi, Series B, Shire, Sialic Acid, Sly Syndrome, Sudden Infant Death Syndrome, triheptanoin, ultra-rare, Ultragenyx
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Oxyrane seeking to advance Pompe Disease programme in 2013
Authored by James Sheppard
Posted in Orphan Drugs and Rare Diseases
Tagged august, Dr Charles Richard, EMA, FDA, Forbion Capital, funding, James Weston, Liftstream, LSDs, LSLraredisease, Lysosomal Storage Disorders, OXY2098-95, Oxyrane, Philip Astley-Sparke, Rare Disease, rare disease day, rare diseases, RDD13, Series D, Susan Flint, USA
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Roche Snags Phase III Agromegaly Drug from Chiasma
Roche have announced they have acquired the rights to a phase III oral Agromegaly drug Octreolin from, Israeli biotech, Chiasma. Agromegaly is a rare progressive disease caused by the over production of growth hormone resulting in severe disfigurement. Agromegaly is estimated to occur in approximately 6 in every 100,000 people.
Posted in M&A Finance and Funding
Tagged Abingworth, Agromegaly, Chiasma, EMA, FDA, MAA, MPM Capital, Octreolin, Orphan Drug, Phase III, Roche, Series D
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