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Sarepta`s Embark Study Part 2 Shows Elevidys Benefits In DMD
27 Jan 2025 //
BUSINESSWIRE
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
FDA Grants Orphan Status To GIVI-MPC For Becker Muscular Dystrophy
13 Jan 2025 //
BUSINESSWIRE
World Orphan Drug Alliance Expands To Canada And Full LATAM Region
10 Jan 2025 //
GLOBENEWSWIRE
Spero Therapeutics Updates Business And Leadership Changes
10 Jan 2025 //
GLOBENEWSWIRE
Rigel Announces R289 Granted Orphan Drug Designation by FDA for MDS
09 Jan 2025 //
PR NEWSWIRE
AscellaHealth Releases Q4 2024 Specialty And Rare Pipeline Digest™
09 Jan 2025 //
GLOBENEWSWIRE
COUR Pharma Gains FDA Orphan Drug Status For CNP-104 In PBC
08 Jan 2025 //
GLOBENEWSWIRE
Atsena Begins Ph I/II Gene Therapy Trial For X-Linked Retinoschisis
08 Jan 2025 //
GLOBENEWSWIRE
ViGeneron Gets Rare Pediatric Designation For VG901 Retinitis Trial
08 Jan 2025 //
GLOBENEWSWIRE
FDA Accepts Agios PYRUKYND NDA For Alpha, Beta-Thalassemia Patients
08 Jan 2025 //
GLOBENEWSWIRE
Avidity Plans BLA Submission for Rare Muscle Diseases by 2025
08 Jan 2025 //
PR NEWSWIRE
AliveGen Gets FDA Orphan Drug Status For ALG-801 In Hypertension
07 Jan 2025 //
BUSINESSWIRE
Orsini, PicnicHealth Partner To Enhance Rare Disease Patient Care
07 Jan 2025 //
PR NEWSWIRE
Telomir Reports Lifespan Restoration In Progeria Preclinical Model
07 Jan 2025 //
ACCESSWIRE
Actuate Receives EMA Orphan Status For Elraglusib In Cancer
07 Jan 2025 //
GLOBENEWSWIRE
Quoin Reports Positive Data On Netherton Syndrome Clinical Study
06 Jan 2025 //
GLOBENEWSWIRE
Tempest Gets FDA Orphan Drug Status For Amezalpat In HCC
06 Jan 2025 //
GLOBENEWSWIRE
NMD670 Gains FDA Orphan Drug Designation For Charcot-Marie-Tooth
06 Jan 2025 //
GLOBENEWSWIRE
Eton Advances Commitment to Rare Disease with Acquisition of Galzin
04 Jan 2025 //
GLOBENEWSWIRE
FDA Grants Orphan Drug Designation to MicuRx for NTM Infections
02 Jan 2025 //
PR NEWSWIRE
Novartis Gene Therapy Benefits Kids with Rare Muscle Disorder
31 Dec 2024 //
PRESS RELEASE
HKBU Aptamer for X-link Hypophosphatemia Receive Orphan Designation
23 Dec 2024 //
PR NEWSWIRE
FDA approves Vertex Pharma`s triple combo cystic fibrosis treatment
21 Dec 2024 //
REUTERS
VC firm pens $608M deal to direct NGM MASH drug to rare disease
20 Dec 2024 //
FIERCE BIOTECH
US FDA approves Ionis Pharma`s genetic disorder drug
20 Dec 2024 //
REUTERS
Vanda Pharma Announces Orphan Drug Designation for VGT-1849A
20 Dec 2024 //
PR NEWSWIRE
Aquestive Receives FDA Orphan Drug Exclusivity for Libervant
19 Dec 2024 //
GLOBENEWSWIRE
Quoin Announces FDA Clearance for QRX003 Netherton Syndrome Study
19 Dec 2024 //
GLOBENEWSWIRE
Sapience Receives Orphan Drug Designation for ST316 in FAP
19 Dec 2024 //
PR NEWSWIRE
Praxis Precision Receives Rare Disease Designation for Relutrigine
18 Dec 2024 //
GLOBENEWSWIRE
Novo Nordisk`s Ozempic faces EU review for potential eye disease connection
18 Dec 2024 //
REUTERS
Europe Grants Orphan Status for Mitapivat in Sickle Cell
18 Dec 2024 //
GLOBENEWSWIRE
Savara Begins BLA Submission for MOLBREEVI in aPAP Treatment
18 Dec 2024 //
BUSINESSWIRE
Hansa Bio`s Positive Results from Ph 2 in Guillain-Barré Syndrome
17 Dec 2024 //
PR NEWSWIRE
Theranexus & Exeltis Announce Agreement for Tx01 in Rare Disorders
17 Dec 2024 //
ACCESSWIRE
FDA Approves Orphan-Drug Designation for Jaguar Health’s Crofelemer
17 Dec 2024 //
ACCESSWIRE
EyeDNA Receives FDA Rare Pediatric Disease Designation for Therapy
17 Dec 2024 //
GLOBENEWSWIRE
Novo`s Ozempic faces scrutiny over potential link to rare eye disease
17 Dec 2024 //
REUTERS
Novo invests $1.2 bln in new rare disease drugs plant in Denmark
17 Dec 2024 //
REUTERS
Ladenburg Hosts Showcase On Jaguar`s Rare Disease Research
13 Dec 2024 //
ACCESSWIRE
XORTX Announces Presentation at the Rare & Genetic Disease Summit
12 Dec 2024 //
GLOBENEWSWIRE
Lumos Pharma & Double Point Complete Tender Offer, Acquisition
12 Dec 2024 //
GLOBENEWSWIRE
n-Lorem Hosts Nano-Rare Colloquium With 750+ Attendees
12 Dec 2024 //
BUSINESSWIRE
Krystal Bio Updates On Gene Therapy For Rare Respiratory Diseases
12 Dec 2024 //
GLOBENEWSWIRE
