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NICE Recommends Novel Gene Therapy for Rare Genetic Brain Disorder https://t.co/hvLzPJmZXa #HEOR #RareDisease #OrphanDrug #Therapeutics #GeneTherapy https://t.co/ayRLXJMLMy
Fri Mar 24 20:30:12 +0000 2023 -
Creative Medical Technology files FDA ODD application for Brittle Type 1 diabetes https://t.co/4r7B4QcdPA #pharma #health #diabetes #FDA #ODD #OrphanDrug https://t.co/Yf3GZoJ72I
Fri Mar 24 14:00:02 +0000 2023 -
#ONOPHARMA USA's #Tirabrutinib Receives #OrphanDrug Designation from the #FDA for the Treatment of Primary #CentralNervousSystemLymphoma https://t.co/ato6weVNpZ
Fri Mar 24 09:15:40 +0000 2023 -
#IchnosSciences Receives #OrphanDrug Designation for First-In-Class #Bispecific (#CD38 #CD47) #Antibody #InnateCell Modulator, #ISB1442 https://t.co/SwOix0qFLM
Wed Mar 22 10:40:26 +0000 2023 -
US FDA grants Zydus Lifesciences’s ‘ZYIL 1’ Orphan Drug Designation @ZydusUniverse | @US_FDA | #orphandrug | #rarediseases | #autoimmunedisease | #pharmaindustry | #drugdevelopment | #fdaapproved | #clinicaltrials | #healthnews Read more at: https://t.co/iLDTMv4ihZ
Wed Mar 22 07:13:40 +0000 2023 -
RT @RareDisease_Adv: Top @US_FDA official touts ‘promise of #GeneTherapy’ at @MDAorg annual #MDAConference. Read @LLuxner's MDA conference coverage: https://t.co/fJ1KsVjewM #RareDisease #OrphanDrug #SpinalMuscularAtrophy #Hemophilia #MedNews #MedEd #DuchenneMuscularDystrophy #HealthEquityInRare
Wed Mar 22 01:01:44 +0000 2023 -
RT @RareDisease_Adv: Top @US_FDA official touts ‘promise of #GeneTherapy’ at @MDAorg annual #MDAConference. Read @LLuxner's MDA conference coverage: https://t.co/fJ1KsVjewM #RareDisease #OrphanDrug #SpinalMuscularAtrophy #Hemophilia #MedNews #MedEd #DuchenneMuscularDystrophy #HealthEquityInRare
Tue Mar 21 20:06:50 +0000 2023 -
RT @RareDisease_Adv: Top @US_FDA official touts ‘promise of #GeneTherapy’ at @MDAorg annual #MDAConference. Read @LLuxner's MDA conference coverage: https://t.co/fJ1KsVjewM #RareDisease #OrphanDrug #SpinalMuscularAtrophy #Hemophilia #MedNews #MedEd #DuchenneMuscularDystrophy #HealthEquityInRare
Tue Mar 21 16:22:12 +0000 2023 -
Top @US_FDA official touts ‘promise of #GeneTherapy’ at @MDAorg annual #MDAConference. Read @LLuxner's MDA conference coverage: https://t.co/fJ1KsVjewM #RareDisease #OrphanDrug #SpinalMuscularAtrophy #Hemophilia #MedNews #MedEd #DuchenneMuscularDystrophy #HealthEquityInRare
Tue Mar 21 15:26:30 +0000 2023 -
The US Food and Drug Administration has approved omaveloxolone #Skyclarys to treat persons 16 years and older with Friedreich’s ataxia @US_FDA @Friedreichsatax #checkrare #rarediseases #FDA #orphandrug https://t.co/uXNC1t9Q9E
Tue Mar 21 15:03:13 +0000 2023 -
#FOREBiotherapeutics Receives #FDA #OrphanDrug Designation for #FORE8394 for the Treatment of Primary #Brain and #CNSMalignancies https://t.co/oY506zuQWv
Tue Mar 21 06:57:22 +0000 2023 -
Zydus granted Orphan Drug Designation by the USFDA for ZYIL1 in treatment of patients with Cryopyrin Associated Periodic Syndrome (CAPS). #Zydus #granted #OrphanDrug #Designation #USFDA #ZYIL1 #treatment #patients #CryopyrinAssociatedPeriodicSyndrome #CAPS https://t.co/TqaX4N69w4
Tue Mar 21 05:01:12 +0000 2023 -
The US Food and Drug Administration #FDA has approved aflibercept #Eylea to treat preterm infants with retinopathy of prematurity. @US_FDA #checkrare #raredisease #orphandrug https://t.co/kM08wEvUCQ
Fri Mar 17 05:00:25 +0000 2023 -
Will you join us in a few hours for our #webinar to find out if the #orphandrug market is slowing down? Click here to register, and we will see you at 3 pm GMT (11 am EST). https://t.co/OnhdQcLaIe https://t.co/W2hR4axM2m
Thu Mar 16 13:03:02 +0000 2023 -
#PTX receives expanded #OrphanDrug Designation for all #TCellLymphomas $PTX https://t.co/Io3zZMIzUF
Thu Mar 16 10:22:39 +0000 2023 -
#EllipsesPharma reported #EP0042 Receives #OrphanDrug Designation from the US #FDA https://t.co/GjbZ4cG72F
Wed Mar 15 10:43:28 +0000 2023 -
RT @RareEndoERN: Don't miss this #orphandrug webinar organized by our ERICA colleagues. A must attend for anyone involved in #raredisease research. Register now: https://t.co/9oT4qs9LKW #ERICA #Endo-ERN #ERN #rarediseases #healthunion #eu4health #EUGlobalHealthStrategy #healthiertogether https://t.co/PT8qkRkvtz
Tue Mar 14 18:14:44 +0000 2023 -
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RT @Share4Rare: 🔎 Chinese researcher and Bietti crystalline dystrophy patient Richard Rui Yang discusses nontransparent science in #raredisease research and its implications for #orphandrug development 👉 https://t.co/i9YZenCd9D @ojrarediseases https://t.co/ZzCNgxRx0p
Mon Mar 13 23:52:03 +0000 2023 -
RT @OptimasticLady: #RareDisease & #OrphanDrug. Identify what works & barriers to accessing ODs in #NorthernIreland; looking for Parents of children (<18 years) with RD experience taking ODs in #NI to have 1 hr interview. If you are interested, Please contact “raredisease@qub.ac.uk”
Mon Mar 13 19:13:14 +0000 2023 -
🔎 Chinese researcher and Bietti crystalline dystrophy patient Richard Rui Yang discusses nontransparent science in #raredisease research and its implications for #orphandrug development 👉 https://t.co/i9YZenCd9D @ojrarediseases https://t.co/ZzCNgxRx0p
Mon Mar 13 11:05:00 +0000 2023 -
In case you missed it - the FDA granted #OrphanDrug designation to belimumab for systemic sclerosis. Learn more: https://t.co/eicFZAiwcc #SystemicSclerosis #Scleroderma
Sun Mar 12 13:05:00 +0000 2023 -
RT @ginaGib68993499: @glen_rouse @iamalsorg @US_FDA @DrCaliff_FDA @FDACBER @statnews @AP_FDAwriter @Newsweek @FedcourtJunkie @edsilverman @DrCaliff_FDA @FDACBER @janschakowsky @rosadelauro #nurOwnNow #ENDALS #StrongEnoughLongEnough #NoAdComNoVoice #AcceleratedApproval #OrphanDrug
Fri Mar 10 23:06:47 +0000 2023 -
RT @ginaGib68993499: @glen_rouse @iamalsorg @US_FDA @DrCaliff_FDA @FDACBER @statnews @AP_FDAwriter @Newsweek @FedcourtJunkie @edsilverman @DrCaliff_FDA @FDACBER @janschakowsky @rosadelauro #nurOwnNow #ENDALS #StrongEnoughLongEnough #NoAdComNoVoice #AcceleratedApproval #OrphanDrug
Fri Mar 10 22:11:15 +0000 2023 -
The @US_FDA granted #OrphanDrug designation for a new induced #pluripotent stem cell therapeutics for #DuchenneMuscularDystrophy (#DMD). Read more here: https://t.co/G7VPPD2Twp #TreatingMD #MuscleDegeneration #MedNews #MedEd https://t.co/0v2fNCZqTP
Fri Mar 10 16:30:03 +0000 2023
