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Sarepta tanks despite positive data on eteplirsen in Duchenne MD

Sarepta tanks despite positive data on eteplirsen in Duchenne MD

11-07-2014

US RNA-based drug developer Sarepta Therapeutics saw its shares plunge, despite the company releasing…

BiotechnologyeteplirsenRare diseasesRegulationResearchSarepta TherapeuticsUSA

Vertex leaps on positive Ph III results with lumacaftor

Vertex leaps on positive Ph III results with lumacaftor

24-06-2014

US biotech firm Vertex Pharmaceuticals saw its share price rocket nearly 52% to $101.20 in extending…

BiotechnologyBusiness FinanceCystic fibrosisHealth Medical PharmaKalydecolumacaftorMedicinePhenolsRare diseasesRegulationResearchVertex Pharmaceuticals

Another $1 million GSK milestone for Isis Pharma

Another $1 million GSK milestone for Isis Pharma

24-06-2014

USA-based Isis Pharmaceuticals said today that it has earned a $1 million milestone payment from GlaxoSmithKline…

FinancialGlaxoSmithKlineIsis PharmaceuticalsISIS-TTR RxPharmaceuticalRare diseasesResearch

UK’s Summit sets up operations in the USA

UK’s Summit sets up operations in the USA

17-06-2014

UK drug discovery firm Summit, which is focused on therapies for Duchenne muscular dystrophy and Clostridium…

Antibiotics and Infectious diseasesBoardroomManagementPharmaceuticalRare diseasesResearchSummit PharmaceuticalsUSA

Santhera muscular dystrophy drug shows success first time in late clinical trial

22-05-2014

For the first time, a drug for Duchenne muscular dystrophy (DMD) – from Swiss drugmaker Santhera Pharmaceuticals…

CatenaPharmaceuticalRare diseasesRaxoneResearchSanthera Pharmaceuticals

Positive Ph III results with Santhera’s Catena/Raxone in Duchenne muscular dystrophy

14-05-2014

Swiss drugmaker Santhera Pharmaceuticals has released positive Phase III, double-blind, placebo-controlled…

CatenaPharmaceuticalRare diseasesRaxoneResearchSanthera Pharmaceuticals

Common ED drug may help treat effects of muscle disease in boys

08-05-2014

A drug typically prescribed for erectile dysfunction or increased pressure in the arteries may help improve…

PharmaceuticalRare diseasesResearchsildenafilTadalafil

Novartis’ Signifor LAR shows superior efficacy in certain acromegaly patients

Novartis’ Signifor LAR shows superior efficacy in certain acromegaly patients

06-05-2014

Swiss drug major Novartis has presented positive results from a pivotal Phase III trial of investigational…

NovartisPharmaceuticalRare diseasesResearchSignifor

Shire 1st-qrt 2014 sales and earnings rocket; lifitegrast disappoints

01-05-2014

Ireland-headquartered drugmaker Shire posted a strong set of financials this afternoon, showing that…

FinancialLifitegrastOphthalmicsPharmaceuticalRare diseasesResearchShire

Amicus Thera leaps on positive migalastat results

29-04-2014

US biotech firm Amicus Therapeutics (has released positive 12- and 24-month data from its first Phase…

Amicus TherapeuticsBiotechnologymigalastatRare diseasesResearch

Merck releases positive Ph IIIb results for Kuvan

24-04-2014

German pharma and life sciences major Merck KGaA announced today that the Phase IIIb SPARK study with…

KuvanMerck KGaAPharmaceuticalRare diseasesResearch

UCB enters strategic alliance with Weill-Cornell in three discovery projects

03-04-2014

Belgian drugmaker UCB has launched a major, three-year strategic research alliance with Weill-Cornell…

MetabolicsPharmaceuticalRare diseasesResearchUCB

Breakthrough therapies for rare diseases command premium pricing

Breakthrough therapies for rare diseases command premium pricing

26-03-2014

The global orphan drugs market presents plenty of opportunities for new drug development – while there…

PharmaceuticalPricingRare diseasesResearch

Promising mid-stage results for Prosensa’s drisapersen in DMD boys

19-03-2014

Shares of Dutch biotech firm Prosensa Holding rose 3.85% to $7.02 after the firm reported encouraging…

BiotechnologydrisapersenGlaxoSmithKlineProsensaRare diseasesResearch

More positive Ph III data for Amgen’s evolocumab

More positive Ph III data for Amgen’s evolocumab

17-03-2014

Leading independent biotech firm Amgen has released more positive clinical trial data with its cholesterol…

AmgenBiotechnologyCardio-vascularevolocumabRare diseasesResearch

Six-year follow-up data from Glybera treated patients

Six-year follow-up data from Glybera treated patients

17-03-2014

Netherlands-based human gene company uniQure and privately-held Italian drugmaker Chiesi Farmaceutici…

BiotechnologyChiesi FarmaceuticiGlyberaRare diseasesResearchuniQure

Marina Biotech to use its therapeutics platform to develop drugs for rare diseases

11-03-2014

US RNAi-based drug discover Marina Biotech says that it plans to broadly apply its RNAi, antisense and…

BiotechnologyFinancialMarina BiotechRare diseasesResearch

Ph III study for Jakavi meets endpoint in patients with polycythemia vera

Ph III study for Jakavi meets endpoint in patients with polycythemia vera

07-03-2014

Swiss pharma major Novartis today announced positive results from a pivotal Phase III trial of Jakavi…

IncyteJakaviNovartisOncologyPharmaceuticalRare diseasesRegulationResearch

Hawaii Biotech awarded $5.5million government grant for botulism therapy

Hawaii Biotech awarded $5.5million government grant for botulism therapy

05-03-2014

Privately-held US biotech company Hawaii Biotech has been awarded a contract to continue developing drugs…

BiotechnologyFinancialHawaii BiotechRare diseasesResearchUSA

ISIS Pharma earns $1 million from GSK for advancing ISIS-TTRRx

03-03-2014

USA-based Isis Pharmaceuticals has earned a $1 million milestone payment from UK pharma giant GlaxoSmithKline…

FinancialGlaxoSmithKlineIsis PharmaceuticalsISIS-TTRRxPharmaceuticalRare diseasesResearch

Which place for orphan drugs in the strategy of pharma companies and payers?

27-02-2014

As the 7th Rare Disease Day will be held on February 28, ALCIMED, an innovation and new business consulting…

Markets & MarketingNovartisPharmaceuticalPricingRare diseasesResearchRocheSanofi

Prothelia and Alexion partner with University of Nevada for ultra-rare neuromuscular disorder

Prothelia and Alexion partner with University of Nevada for ultra-rare neuromuscular disorder

17-02-2014

Privately-held USA-based Prothelia and the University of Nevada, Reno, have entered into strategic agreements…

Alexion PharmaceuticalsMergers & AcquisitionsPharmaceuticalProtheliaRare diseasesResearch

Sanofi renews alliance with Regulus and ups investment by $10 million

Sanofi renews alliance with Regulus and ups investment by $10 million

05-02-2014

US pharma company Regulus Therapeutics has renewed its strategic alliance with French drug major Sanofi…

FinancialFranceOncologyPharmaceuticalRare diseasesRegulus TherapeuticsResearchSanofiUSA

UCB launches online search for rare phenotypes

UCB launches online search for rare phenotypes

28-01-2014

Belgian drugmaker UCB (Euronext Brussels: UCB) has launched an online search to find rare phenotypes…

GeneticsGlobalPharmaceuticalRare diseasesResearchUCB

1 to 25 of 127 results

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Eleven Biotherapeutics

Eleven Biotherapeutics

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