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Catalyst Pharma updates on Ph III Firdapse study in LEMS

13-08-2014

US rare diseases focussed biotech firm Catalyst Pharmaceutical Partners provided an update on the progress…

BiotechnologyCatalyst Pharmaceutical PartnersFirdapseLambert-Eaton myasthenic syndromeRare diseasesRegulationResearchTreatment of Lambert-Eaton myasthenic syndromeUSA

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

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

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

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

BioLineRx gains orphan status for novel stem cell mobilization treatment

BioLineRx gains orphan status for novel stem cell mobilization treatment

23-01-2014

Israeli drug developer BioLineRx has received notice from the US Food and Drug Administration confirming…

BioLineRxBiotechnologyBL-8040Rare diseasesRegulationResearch

Genzyme to gain rights to certain Alnylam rare disease pipeline products

Genzyme to gain rights to certain Alnylam rare disease pipeline products

13-01-2014

French drug major Sanofi’s US biotech subsidiary Genzyme and Alnylam Pharmaceuticals have significantly…

Alnylam PharmaceuticalsBiotechnologyGenzymeLicensingMergers & AcquisitionspatisiranRare diseasesResearchSanofi

arGEN-X reaches two milestone payments in Shire deal and signs research agreement with Boehringer

13-01-2014

Belgium-based biotech firm arGEN-X has reached two important success milestones in its SIMPLE Antibody…

arGEN-XBelgiumBiotechnologyBoehringer IngelheimFinancialGeneticsLicensingRare diseasesResearchShire

Agilis Biotherapeutics and Intrexon collaborate on rare genetic disease

01-01-2014

US synthetic biology companies Agilis Biotherapeutics and Intrexon have entered an exclusive channel…

Agilis BiotherapeuticsBiotechnologyBoardroomFinancialIntrexonManagementRare diseasesResearch

FDA grants priority review for Genzyme’s Cerdelga

FDA grants priority review for Genzyme’s Cerdelga

11-12-2013

The US Food and Drug Administration has granted Sanofi (Euronext: SAN) and its subsidiary Genzyme a six-month…

BiotechnologyeliglustatGenzymeNorthern EuropeRare diseasesResearch

Progenics relaunches registrational trial of Azedra in pheochromocytomas

25-11-2013

USA-based Progenics Pharmaceuticals says it is resuming a Phase IIb clinical study of safety and efficacy…

AzedraBiotechnologyOncologyProgenics PharmaceuticalsRare diseasesResearch

Sarepta plunges as FDA questions eteplirsen trial results

Sarepta plunges as FDA questions eteplirsen trial results

12-11-2013

Shares of US RNA-based drug developer Sarepta Therapeutics plummeted in pre-market trading on November…

BiotechnologydrisaperseneteplirsenGlaxoSmithKlineNorth AmericaRare diseasesRegulationResearchSarepta Therapeutics

Bone Therapeutics’ pivotal Ph III osteonecrosis trial with PREOB hits a new milestone

Bone Therapeutics’ pivotal Ph III osteonecrosis trial with PREOB hits a new milestone

07-11-2013

Closely-held Belgian regenerative medicines company Bone Therapeutics says that its Phase III pivotal…

BiotechnologyBone TherapeuticsPreobRare diseasesResearch

Vertex outlines progress on cystic fibrosis drug development

17-10-2013

USA-based biotech firm Vertex Pharmaceuticals provided a comprehensive update on recent progress in its…

BiotechnologyKalydecolumacaftorRare diseasesResearchVertexVX-661

Galapagos touts superior potentiator series for cystic fibrosis

17-10-2013

Belgian biotech firm Galapagos has developed a series of novel potentiators in its cystic fibrosis program,…

BiotechnologyGalapagosRare diseasesResearch

Isis Pharma earns $10 million milestone in deal with Biogen

16-10-2013

Isis Pharmaceuticals says that it has earned a $10 million milestone payment from fellow USA-based biotech…

Biogen IdecBiotechnologyFinancialIsis PharmaceuticalsISIS-DMPKrxRare diseasesResearch

Kyowa Hakko Kirin collaborates with Ultragenyx on KRN23

06-09-2013

Japanese mid-sized drugmaker Kyowa Hakko Kirin (TYO: 4151) has entered into a collaboration and license…

BiotechnologyKRN23Kyowa Hakko KirinLicensingPharmaceuticalRare diseasesResearchUltragenyx

iPierian secures funding for launch of spin-out, True North Therapeutics

05-09-2013

US biotech firm iPierian says that a new financing of $30 million has been secured to continue to advance…

BiotechnologyFinancialiPierianIPN007NeurologicalRare diseasesResearchTrue North Therapeutics

Shire and Santaris extend strategic alliance

23-08-2013

Danish privately held clinical-stage biopharmaceutical company Santaris Pharma says that its long term…

BiotechnologyLicensingPharmaceuticalRare diseasesResearchSantaris PharmaShire

Deal between Ensemble and Alexion will target ultra-rare disorders

18-07-2013

USA-based drug discovery firm Ensemble Therapeutics has announced the initiation of a drug discovery…

Alexion PharmaceuticalsBiotechnologyEnsemble TherapeuticsNorth AmericaPharmaceuticalRare diseasesResearch

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