Biotechnology - Rare diseases

Filter

Current filters:

Rare diseases

Popular Filters

1 to 25 of 153 results

Pharming and Salix gain US approval for Ruconest

Pharming and Salix gain US approval for Ruconest

17-07-2014

Netherlands-based Pharming Group and Salix Pharmaceuticals have received approval from the US Food and…

BiotechnologyPharmingRare diseasesRegulationRuconestSalix PharmaceuticalsUSA

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

Baxter buys AesRx, gaining rights to investigational SCD drug

Baxter buys AesRx, gaining rights to investigational SCD drug

10-07-2014

US drugmaker Baxter International said yesterday that it has acquired AesRx, a private US biopharmaceutical…

Aes-103AesRxBaxter InternationalBiotechnologyHematologyMergers & AcquisitionsRare diseasesUSA

US orphan status for Mirati’s mocetinostat and Alexion’s Soliris

US orphan status for Mirati’s mocetinostat and Alexion’s Soliris

17-06-2014

USA-based Mirati Therapeutics has been granted Orphan Drug designation by the US Food and Drug Administration…

BiotechnologyOncologyRare diseasesRegulation

uniQure signs up Medison to distribute Glybera in Israel

uniQure signs up Medison to distribute Glybera in Israel

29-05-2014

Netherlands-based human gene therapy firm uniQure has entered an exclusive distribution agreement with…

BiotechnologyGlyberaLicensingMedison PharmaRare diseasesRest of the WorlduniQure

CHMP changes mind on PTC Therapeutics’ Duchenne drug Translarna, giving a positive opinion

25-05-2014

In something of a surprise for PTC Therapeutics and observers, the European Medicines Agency’s advisory…

BiotechnologyEuropePTC TherapeuticsRare diseasesRegulationTranslarna

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

US FDA clears pathway for Sarepta’s eteplirsen filing by year end

22-04-2014

US RNA-based drug developer Sarepta Therapeutics it plans to submit a New Drug Application to the US…

BiotechnologyeteplirsenNorth AmericaRare diseasesRegulationSarepta TherapeuticsUSA

SOBI assumes direct responsibility for rare disease drug Orfadin in the USA and Canada

SOBI assumes direct responsibility for rare disease drug Orfadin in the USA and Canada

01-04-2014

Biotech firm Swedish Orphan Biovitrum has assumed direct responsibility for the distribution of its proprietary…

BiotechnologyMarkets & MarketingNorthern EuropeOrfadinRare diseasesSobi

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

UK’s BIA finds government unwilling to set price cap for orphan disease treatment

UK’s BIA finds government unwilling to set price cap for orphan disease treatment

11-03-2014

The majority of Members of Parliament in the UK believe there should not be a maximum price per patient…

BiotechnologyPoliticsPricingRare diseasesUK

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

FDA backs Kalydeco for eight additional mutations that cause cystic fibrosis

22-02-2014

The US Food and Drug Administration approved biotech firm Vertex Pharmaceuticals’ supplemental New…

BiotechnologyKalydecoNorth AmericaRare diseasesRegulationUSAVertex

FDA approves Northera for neurogenic orthostatic hypotension

FDA approves Northera for neurogenic orthostatic hypotension

19-02-2014

Shares of US biotech firm Chelsea Therapeutics soared 38% to $6.75 in after-hours trading yesterday,…

BiotechnologyChelsea TherapeuticsDainippon Sumitomo PharmaNorth AmericaNortheraRare diseasesRegulationUSA

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

BioMarin buys rights to Repligen preclinical compounds

23-01-2014

BioMarin Pharmaceuticals has entered into an agreement to purchase from fellow USA-based Repligen Corp…

BioMarin PharmaceuticalBiotechnologyLicensingRare diseasesRepligen

GlaxoSmithKline returns Duchenne drug rights to Prosensa

GlaxoSmithKline returns Duchenne drug rights to Prosensa

13-01-2014

UK pharma giant GlaxoSmithKline says that its partner, Dutch biotech firm Prosensa has regained all rights…

BiotechnologydrisapersenGlaxoSmithKlineLicensingProsensaRare diseases

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

1 to 25 of 153 results

Back to top