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Genethon Marks Rare Disease Day With Advances In Gene Therapies
27 Feb 2025 //
BUSINESSWIRE
Regeneron gene therapy improves hearing in 10 children
25 Feb 2025 //
PRESS RELEASE
FDA Grants Priority Review To Precigen`s PRGN-2012 For RRP
25 Feb 2025 //
PR NEWSWIRE
GRIN Therapeutics gets FDA Breakthrough Therapy for Radiprodil
25 Feb 2025 //
PR NEWSWIRE
YolTech Therapeutics: Positive Data of YOLT-203 for PH1
25 Feb 2025 //
PR NEWSWIRE
Sterotherapeutics Begins Phase 2 Trial for Cushing`s Syndrome
24 Feb 2025 //
PR NEWSWIRE
FDA Approves First Treatment For Cerebrotendinous Xanthomatosis
21 Feb 2025 //
PR NEWSWIRE
FDA Priority Review To PTC’s Vatiquinone For Friedreich’s Ataxia
19 Feb 2025 //
PR NEWSWIRE
Biogen and Stoke Strike Dravet Syndrome Co-Development Deal
19 Feb 2025 //
BIOSPACE
SpringWorks Therapeutics and Jennifer Fisher Encourage Action Aga
19 Feb 2025 //
PR NEWSWIRE
FDA Grant Priority Review To Ultragenyx`s BLA For UX111 In MPS IIIA
18 Feb 2025 //
GLOBENEWSWIRE
FDA approves Ono`s rare tumor drug from $2.4B Deciphera buyout
15 Feb 2025 //
FDA
Benitec’s BB-301 Ph1b/2a Data Accepted For MDA Conference
14 Feb 2025 //
GLOBENEWSWIRE
Sarepta Announces Inaugural $600 M Senior Secured Credit Facility
14 Feb 2025 //
BUSINESSWIRE
Ensoma Receives Pediatric & Orphan Designations for EN-374 in CGD
13 Feb 2025 //
BUSINESSWIRE
Agios’ Ph 3 Activate-Kids Hits Primary Endpoint In PK Deficiency
13 Feb 2025 //
GLOBENEWSWIRE
Elixirgen Gets RMAT Designation for EXG-34217 in Telomere Disorders
13 Feb 2025 //
GLOBENEWSWIRE
Recordati 2024 Preliminary Results: Revenue +12.4%, EBITDA +12.5%
13 Feb 2025 //
GLOBENEWSWIRE
US FDA approves SpringWorks` genetic disorder drug
12 Feb 2025 //
PRESS RELEASE
Rezolute Reports Q2 Fiscal 2025 Financial Results & Update
12 Feb 2025 //
GLOBENEWSWIRE
FDA approves Roche’s Evrysdi tablet as first & only tablet for SMA
12 Feb 2025 //
GLOBENEWSWIRE
Opna Bio Gets Orphan Drug Status For OPN-6602 In Multiple Myeloma
12 Feb 2025 //
BUSINESSWIRE
Biodexa’s FAP Drug Gets FDA Fast Track After Positive Ph 2 Results
12 Feb 2025 //
ACCESSWIRE
Palvella Expands Ph 3 Trial For Microcystic Lymphatic Malformations
10 Feb 2025 //
GLOBENEWSWIRE
INmune Plans BLA for CORDStrom in Dystrophic Epidermolysis Bullosa
10 Feb 2025 //
GLOBENEWSWIRE
Hemab Therapeutics Presents on HMB-001 for Glanzmann Thrombasthenia
07 Feb 2025 //
PR NEWSWIRE
AceLink Presents Results for AL01211 in Fabry Disease Patients
07 Feb 2025 //
BUSINESSWIRE
X4 Pharma`s Restructure for Mavorixafor in Chronic Neutropenia
06 Feb 2025 //
GLOBENEWSWIRE
FDA Grants Orphan Drug Designation to Ariceum`s Cancer Therapy
06 Feb 2025 //
PHARMAWEB
Arbor Biotechnologies Announces FDA Designations for ABO-101
05 Feb 2025 //
GLOBENEWSWIRE
Ultragenyx Shows UX111 Therapy Improves Sanfilippo Syndrome Type A
05 Feb 2025 //
GLOBENEWSWIRE
Mitem Acquires FLISINT Specialty From Sanofi for Rare Diseases
04 Feb 2025 //
BUSINESSWIRE
Shionogi & Guardian Launch 1st Drug Study for Jordan’s Syndrome
04 Feb 2025 //
BUSINESSWIRE
Cumberland Reports Ph 2 Breakthrough in DMD Heart Disease Trial
04 Feb 2025 //
PR NEWSWIRE
Quoin Pharma Launches Campaign for Netherton Syndrome Awareness
04 Feb 2025 //
GLOBENEWSWIRE
X4 Pharma Announces Inducement Grants Under NasdaqLR 5635(c)(4)
03 Feb 2025 //
GLOBENEWSWIRE
Chiesi Rare Diseases Announces Presentations at WORLDSymposium™
03 Feb 2025 //
PR NEWSWIRE
Zevra Therapeutics to Present at the 21st Annual WORLDSymposium
30 Jan 2025 //
GLOBENEWSWIRE
Zevra Therapeutics to Participate at Upcoming Investor Conferences
29 Jan 2025 //
GLOBENEWSWIRE
SEED Recieves Orphan Drug Designations For RBM39 Degrader
28 Jan 2025 //
GLOBENEWSWIRE
March Bio FDA Orphan Drug Designation For MB-105 In T-Cell Lymphoma
28 Jan 2025 //
GLOBENEWSWIRE
Sarepta`s Embark Study Part 2 Shows Elevidys Benefits In DMD
27 Jan 2025 //
BUSINESSWIRE
Quince’s EryDex Long-Term Safety Data Publish in Neurology Journal
27 Jan 2025 //
BUSINESSWIRE
X4 Pharma’s Mavorixafor MAA Validated By EMA For WHIM Syndrome
24 Jan 2025 //
GLOBENEWSWIRE
Zydus receives FDA Orphan Drug Designation for ALS
23 Jan 2025 //
ECONOMICTIMES
Larimar Doses Adolescents In Study For Friedreich’s Ataxia
23 Jan 2025 //
GLOBENEWSWIRE
Quoin Shows QRX003 Effectiveness In Treating Netherton Syndrome
23 Jan 2025 //
GLOBENEWSWIRE
Cancer Research UK, Cytovation Collaborate On Rare Cancer Treatment
23 Jan 2025 //
GLOBENEWSWIRE
Curant Launches Curant Rare For Rare & Orphan Disease Care
23 Jan 2025 //
BUSINESSWIRE
FDA Grants Orphan Status To Tikun’s Rare FD Treatment
23 Jan 2025 //
CONTRACTPHARMA
Zai Lab Receives Orphan Designation For ZL-1310 (DLL3 ADC) In SCLC
22 Jan 2025 //
BUSINESSWIRE
Jaguar Health Doses 1st Patient In Evaluating Crofelemer For MVID
22 Jan 2025 //
YAHOOFINANCE
KalVista Submits NDA, Gets Orphan Status For Sebetralstat In Japan
21 Jan 2025 //
BUSINESSWIRE
Biocon urges gov to exempt cancer, rare-disease drugs from tax
20 Jan 2025 //
REUTERS
FDA declines to approve Atara Biotherapeutics` cancer therapy
17 Jan 2025 //
PRESS RELEASE
Bio-Thera Publishes PH 1 Data On BAT4406F For Neuromyelitis Optica
14 Jan 2025 //
PR NEWSWIRE
PacBio, Radboud UMC Advance Rare Disease Diagnostic With Sequencing
14 Jan 2025 //
GLOBENEWSWIRE
Quoin Pharma Reports Data From Pediatric Netherton Syndrome Study
14 Jan 2025 //
GLOBENEWSWIRE
NGGT Publishes Early Clinical Trial Results for BCD Treatment
14 Jan 2025 //
PR NEWSWIRE
Agios Announces 2025 Milestones For Rare Disease Portfolio
13 Jan 2025 //
GLOBENEWSWIRE
