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FDA approves Vertex Pharma`s triple combo cystic fibrosis treatment
21 Dec 2024 //
REUTERS
US FDA approves Ionis Pharma`s genetic disorder drug
20 Dec 2024 //
REUTERS
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
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
Eversana Recognized For Specialty Pharmacy, Rare Disease Care
12 Dec 2024 //
PR NEWSWIRE
Acadia Pharma Closes Sale of Pediatric Disease Priority Review
11 Dec 2024 //
BUSINESSWIRE
Protara Announces Closing of $100 Million Public Offering
11 Dec 2024 //
GLOBENEWSWIRE
Govt prescribes faster access to medicines for rare diseases
11 Dec 2024 //
ECONOMICTIMES
Ocugen CEO to Present at Oppenheimer Movers in Rare Disease Summit
11 Dec 2024 //
GLOBENEWSWIRE
Pilatus Biosciences Secures FDA Orphan Drug Designation for PLT012
10 Dec 2024 //
PR NEWSWIRE
Stoke Therapeutic Presents Data On Zorevunersen For Dravet Syndrome
06 Dec 2024 //
BUSINESSWIRE
Edgewise Issues Statement on Relationship with Dr. Han Phan
05 Dec 2024 //
BUSINESSWIRE
Roivant to stop lung disease drug development after trial failure
04 Dec 2024 //
REUTERS
AlveoGene’s Inhaled Therapy AVG-002 Gets FDA Orphan Designation
03 Dec 2024 //
GLOBENEWSWIRE
PIF Gets FDA Pediatric Designation for sJIA Small Molecule
03 Dec 2024 //
BUSINESSWIRE
FDA Grants Orphan Designation to EXG110 for Fabry Disease
03 Dec 2024 //
BUSINESSWIRE
Satellos Joins Oppenheimer Rare Disease Summit
03 Dec 2024 //
BUSINESSWIRE
NeuroPace Completes Lennox-Gastaut Syndrome Study
03 Dec 2024 //
GLOBENEWSWIRE
Novo Nordisk Highlights Rare Blood Disorders at ASH 2024
03 Dec 2024 //
PR NEWSWIRE
Genethon, Hansa Begin Phase 2 for Imlifidase in Crigler-Najjar
03 Dec 2024 //
PR NEWSWIRE
Know Rare Launches HealthStoryAI App for Rare Disease Patients
03 Dec 2024 //
GLOBENEWSWIRE
Restem`s FDA Orphan Drug Designation for Polymyositis Treatment
03 Dec 2024 //
GLOBENEWSWIRE
FDA Grants Orphan Drug Designation to Rezolute for Hypoglycemia
03 Dec 2024 //
GLOBENEWSWIRE
Unnatural Products Announces BridgeBio Option for Macrocyclic
03 Dec 2024 //
GLOBENEWSWIRE
Sparsentan Receives Orphan Drug Designation for IgA Nephropathy
03 Dec 2024 //
PR NEWSWIRE
Chemomab to Participate in Oppenheimer’s Rare Disease Summit
03 Dec 2024 //
GLOBENEWSWIRE
Elevar offers $500M biobucks for Relay`s rare cancer asset
03 Dec 2024 //
FIERCE BIOTECH
Avenacy to Attend 2024 ASHP Midyear Clinical Meeting and Exhibition
02 Dec 2024 //
BUSINESSWIRE
Inozyme Pharma to Participate in Piper Sandler Healthcare Conf.
27 Nov 2024 //
GLOBENEWSWIRE
CervoMed Gets Orphan Drug Status for Neflamapimod in FTD
27 Nov 2024 //
GLOBENEWSWIRE
Merck`s therapy for rare lung condition to help reduce risk of death
26 Nov 2024 //
REUTERS
BridgeBio Selects PANTHERx® for Attruby™ Distribution
26 Nov 2024 //
PR NEWSWIRE
Soleno Therapeutics Announces FDA Review Extension for DCCR Tablets
26 Nov 2024 //
GLOBENEWSWIRE
X4 Pharma to Participate in Piper Sandler Healthcare Conference
26 Nov 2024 //
GLOBENEWSWIRE
Zevra Therapeutics to Participate at Upcoming Investor Conferences
26 Nov 2024 //
GLOBENEWSWIRE
Eton Pharmaceuticals Acquires U.S. Rights to Amglidia Suspension
25 Nov 2024 //
GLOBENEWSWIRE
US FDA approves BridgeBio`s drug for rare heart condition
23 Nov 2024 //
PRESS RELEASE
FDA Grants Orphan Drug Status to LBL-024 for Neuroendocrine Cancer
22 Nov 2024 //
PR NEWSWIRE
Zevra Therapeutic Announces US Commercial Availability of Miplyffa
21 Nov 2024 //
GLOBENEWSWIRE
Science 37 Doubles US Enrollment for GSK Phase 3 Rare Disease Trial
21 Nov 2024 //
GLOBENEWSWIRE
Plant compound restores sensation in mice with nerve disorder
20 Nov 2024 //
FIERCE BIOTECH
Capricor Granted Orphan Drug And ATMP Status For Deramiocel
20 Nov 2024 //
GLOBENEWSWIRE
ImmuneSensor Receives Orphan Drug & Rare Disease Designations
20 Nov 2024 //
GLOBENEWSWIRE
Ocugen Receives Orphan Drug Designation From EMA For OCU410ST
20 Nov 2024 //
GLOBENEWSWIRE
STUDY LINKS RARE GENETIC MUTATION TO SEVERE NEUROLOGICAL DISORDER
20 Nov 2024 //
PHARMAWEB
GeneDx Launches GeneDx Discover for Rare Disease Drug Discovery
19 Nov 2024 //
BUSINESSWIRE
Ractigen Wins FDA Orphan Drug Designation for RAG-21 in ALS
19 Nov 2024 //
PR NEWSWIRE
Siren Biotech Reveals SRN-101 as Lead for High-Grade Gliomas
18 Nov 2024 //
GLOBENEWSWIRE
Alkeus`s FDA Designations for Gildeuretinol in Stargardt Disease
18 Nov 2024 //
GLOBENEWSWIRE
Children`s Hospital Announces Gene Therapy Results for Danon
18 Nov 2024 //
PR NEWSWIRE
Vir Biotech Receives EMA Orphan Drug Opinion for Tobevibart
18 Nov 2024 //
BUSINESSWIRE
Eisai ends lorcaserin development in Dravet syndrome
18 Nov 2024 //
FIERCE PHARMA
