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AstraZeneca`s Fasenra Approved in the US for Rare Blood Vessel Disease
19 Sep 2024 //
CONTRACTPHARMA
FDA scraps adcomm for Applied`s rare disease drug ahead of November decision
18 Sep 2024 //
FIERCE BIOTECH
AstraZeneca`s Fasenra Approved in the US for Rare Blood Vessel Disease
18 Sep 2024 //
CONTRACT PHARMA
Papillon Therapeutics Receives Orphan Drug Designation For Friedreich`s Ataxia
17 Sep 2024 //
BUSINESSWIRE
Ainos Plans Taiwan Clinical Study For VELDONA As Treatment For Oral Warts In HIV
17 Sep 2024 //
ACCESSWIRE
Fulcrum Therapeutics Reports Inducement Grants Under Nasdaq Rule
13 Sep 2024 //
GLOBENEWSWIRE
FDA Grants RPD Designation To Senhwa`s Silmitasertib For Neuroblastoma
12 Sep 2024 //
PR NEWSWIRE
Vironexis exits stealth with $26M, debuts rare disease gene therapies
12 Sep 2024 //
FIERCE BIOTECH
Mereo BioPharma To Participate In Cantor Global Healthcare Conference
12 Sep 2024 //
GLOBENEWSWIRE
Actuate Gets FDA Orphan Drug Designation For Elraglusib In Sarcomas
11 Sep 2024 //
GLOBENEWSWIRE
Agios` Tebapivat Gets FDA Orphan Drug Status For Myelodysplastic Syndromes
11 Sep 2024 //
GLOBENEWSWIRE
Oxford-Harrington Centre Adds UK Health Policy Leader To Advisory Council
11 Sep 2024 //
GLOBENEWSWIRE
Ascendis Pharma Gets US Orphan Drug Exclusivity For YORVIPATH
11 Sep 2024 //
GLOBENEWSWIRE
AISA Pharma Gets FDA Orphan Drug Status For AISA-021 In Systemic Sclerosis
09 Sep 2024 //
PR NEWSWIRE
Savara Presents IMPALA-2 Trial Data On Molgramostim For aPAP At ERS
08 Sep 2024 //
BUSINESSWIRE
Zevra Presents New Data On Arimoclomol And OLPRUVA At SSIEM Symposium
06 Sep 2024 //
GLOBENEWSWIRE
Savara Announces New Employment Inducement Grant
06 Sep 2024 //
BUSINESSWIRE
Europe Grants Orphan Drug Designation for Vivet`s Gene Therapy for CTX
05 Sep 2024 //
PR NEWSWIRE
Dion Foundation and Atamyo Partner to Expand US Trial of ATA-200 for LGMD2C/R5
04 Sep 2024 //
BUSINESSWIRE
Skyline Therapeutics` SKG1108 Gene Therapy Receives FDA Orphan Status
03 Sep 2024 //
PR NEWSWIRE
The FDA Needs a Dedicated ‘Center for Rare Diseases’
03 Sep 2024 //
BIOSPACE
X4 Pharmaceuticals to Participate in Upcoming September Investor Conferences
28 Aug 2024 //
GLOBENEWSWIRE
Vaderis Drug Reduces Nosebleeds In Rare Blood Vessel Disorder Trial
28 Aug 2024 //
FIERCE BIOTECH
SciSparc Grants Global Rights For Pain Therapy In $6 Million Agreement
28 Aug 2024 //
GLOBENEWSWIRE
CAMP4 Therapeutics Gets Rare Pediatric Disease Designation For CMP-CPS-001
27 Aug 2024 //
GLOBENEWSWIRE
EC Authorizes Avanzanite Bioscience`s Orphan Drug AKANTIOR
27 Aug 2024 //
BUSINESSWIRE
RedHill`s Opaganib Gets FDA Orphan Drug Status For Childhood Neuroblastoma
26 Aug 2024 //
PR NEWSWIRE
Ractigen`s RAG-18 Gets FDA Orphan Drug Designation For DMD/BMD
22 Aug 2024 //
PR NEWSWIRE
Upsher-Smith Selects PANTHERx Rare For Distribution Of TORPENZ
20 Aug 2024 //
PR NEWSWIRE
Roche Regains Reputation Crown Among Rare Disease Patient Groups
20 Aug 2024 //
FIERCE PHARMA
Opus Genetics` OPGx-LCA5 Gets FDA Rare Pediatric Disease Designation
20 Aug 2024 //
GLOBENEWSWIRE
ReAlta`s RLS-0071 Gets FDA Orphan Drug And Fast Track Designations
19 Aug 2024 //
BUSINESSWIRE
Pantherv Rare Partners With Gilead For Distribution Of LIVDELZI®
16 Aug 2024 //
PR NEWSWIRE
Atsena Therapeutics Gets FDA Rare Pediatric Disease Designation For ATSN-201
14 Aug 2024 //
GLOBENEWSWIRE
Wave Life Sciences Gets FDA Rare Pediatric Status For DMD Treatment
12 Aug 2024 //
GLOBENEWSWIRE
Galien Foundation Announces 2024 Prix Galien USA Nominees
09 Aug 2024 //
PR NEWSWIRE
US FDA approves Citius` therapy for rare blood cancer
09 Aug 2024 //
REUTERS
Zevra Therapeutics Announces Proposed Public Offering of Common Stock
08 Aug 2024 //
GLOBENEWSWIRE
Satellos Receives Rare Pediatric Disease Designation For SAT-3247
08 Aug 2024 //
BUSINESSWIRE
Kyowa Kirin, NewBridge To Improve Rare Disease Medicine Access In MENA
08 Aug 2024 //
BIOSPECTRUM ASIA
Senhwa`s Silmitasertib Gets FDA Nod For Pediatric Cancer Combo Trial
06 Aug 2024 //
PR NEWSWIRE
SELLAS Receives EMA Orphan Drug Designation For SLS009
06 Aug 2024 //
GLOBENEWSWIRE
Upsher-Smith Launches TORPENZ™ Tablets For Rare Diseases
05 Aug 2024 //
PR NEWSWIRE
META-001-PH Granted Rare Pediatric Disease Designation By FDA
05 Aug 2024 //
PR NEWSWIRE
Clinical intuition` led FDA advisors to back Zevra`s rare disease med
05 Aug 2024 //
FIERCE BIOTECH
Rezolute`s IND For RZ358 Phase 3 Study Cleared By FDA
05 Aug 2024 //
GLOBENEWSWIRE
Attralus Gets Breakthrough Therapy For Pan-Amyloid Diagnostic PET Imaging
05 Aug 2024 //
GLOBENEWSWIRE
Rare Pediatric Disease Voucher Drugs` Revenue Equals Brand Drugs
02 Aug 2024 //
RAPS
FDA Questions Evidence For Zevra`s Rare Disease Therapy Pre-Adcomm
02 Aug 2024 //
BIOSPACE
FDA Panel Votes Favorably On Arimoclomol For Niemann-Pick Disease Type C
02 Aug 2024 //
GLOBENEWSWIRE
Amylyx`s AMX0035 Gets EU Orphan Drug Status For Wolfram Syndrome
02 Aug 2024 //
BUSINESSWIRE
FDA Grants Orphan Drug Designation To Cellectis` CLLS52 For ALL
01 Aug 2024 //
GLOBENEWSWIRE
X4 Pharmaceuticals Announces Inducement Grants
01 Aug 2024 //
GLOBENEWSWIRE
Elevar Therapeutics Gets EU Orphan Drug Designation For HCC Therapy
01 Aug 2024 //
GLOBENEWSWIRE
Agios Reports Business Highlights and Second Quarter 2024 Financial Results
01 Aug 2024 //
GLOBENEWSWIRE
Agios Announces Phase 3 ACTIVATE-KidsT Study Results
01 Aug 2024 //
GLOBENEWSWIRE
Vertex, beaten by rare disease again, drops 2 assets on growing discard pile
01 Aug 2024 //
FIERCE BIOTECH
The Siegel Rare Neuroimmune Association Kicks Off Awareness Campaign
31 Jul 2024 //
ACCESSWIRE
Theriva Biologics Gets FDA Rare Pediatric Drug Designation For VCN-01
31 Jul 2024 //
GLOBENEWSWIRE
Charles River Collaborates With FOXG1 Research Foundation
30 Jul 2024 //
BUSINESSWIRE
