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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
Children`s Hospital Announces Gene Therapy Results for Danon
18 Nov 2024 //
PR NEWSWIRE
Eisai ends lorcaserin development in Dravet syndrome
18 Nov 2024 //
FIERCE PHARMA
AlveoGene Gets FDA Rare Pediatric Disease Designation for AVG-002
15 Nov 2024 //
GLOBENEWSWIRE
Perspectum Awarded FDA Grant for Rare Disease Biomarker Study
14 Nov 2024 //
BUSINESSWIRE
Amgen Presents New Data on Rare Inflammatory Diseases at ACR 2024
14 Nov 2024 //
PR NEWSWIRE
Biocodex Announces DIACOMIT® Oral Suspension for Dravet Syndrome
14 Nov 2024 //
PR NEWSWIRE
Soligenix Launches Phase 2 Trial for SGX945 in Behçet`s Disease
14 Nov 2024 //
PR NEWSWIRE
FDA approves PTC Therapeutics` gene therapy for ultra-rare disorder
14 Nov 2024 //
REUTERS
X4 Pharma Reports Positive Phase 2 Results in Chronic Neutropenia
13 Nov 2024 //
GLOBENEWSWIRE
Healx Partners with Sanofi to Explore Rare Disease AI Drug Discovery
13 Nov 2024 //
BUSINESSWIRE
NurExone Secures EMA Orphan Status for ExoPTEN in Spinal Injury
13 Nov 2024 //
GLOBENEWSWIRE
Oranda Therapeutics Launches Series A Funding for Rare Treatments
12 Nov 2024 //
BUSINESSWIRE
Caliway Gets EMA Orphan Designation for CBL-514 in Dercum’s Disease
12 Nov 2024 //
PR NEWSWIRE
Avidity Announces New Precision Cardiology Development Candidates
12 Nov 2024 //
PR NEWSWIRE
Merck, AstraZeneca Announce Phase 3 Win for Cancer Drug
12 Nov 2024 //
FIERCE BIOTECH
Merck KGaA plans China submission after win for tumor drug
12 Nov 2024 //
ENDPTS
Zevra to Present Data on Niemann-Pick Disease Type C at CNS
11 Nov 2024 //
GLOBENEWSWIRE
Upsher-Smith Presents Rare Disease Portfolio at CNS Annual Meeting
11 Nov 2024 //
PR NEWSWIRE
Neurizon Receives Orphan Drug Status for NUZ-001 in Europe
11 Nov 2024 //
PR NEWSWIRE
Relief Announces Results of RLF-TD011 in Epidermolysis Bullosa
11 Nov 2024 //
ACCESSWIRE
ARTHEx Bio Receive FDA Rare Pediatric Designation for ATX-01 in DM1
07 Nov 2024 //
PR NEWSWIRE
Mustang Bio Granted Orphan Drug Status for MB-108 in Glioma
07 Nov 2024 //
GLOBENEWSWIRE
Zevra Therapeutics to Attend Guggenheim Healthcare Conference
06 Nov 2024 //
GLOBENEWSWIRE
Cumberland Pharmaceuticals Receives FDA Designations for Duchenne
06 Nov 2024 //
PR NEWSWIRE
Leads Biolabs Gets FDA Orphan Drug Status for LBL-034 in Myeloma
06 Nov 2024 //
PR NEWSWIRE
Agios Presents New Data on Mitapivat & Tebapivat at ASH 2024
05 Nov 2024 //
GLOBENEWSWIRE
Quoin Pharma Initiates Pediatric Testing for Netherton Syndrome
05 Nov 2024 //
GLOBENEWSWIRE
Arrowhead targets rare disease patients with awareness campaign
05 Nov 2024 //
FIERCE PHARMA
Acadia Sells Rare Pediatric Disease Priority Review Voucher for $150M
05 Nov 2024 //
BUSINESSWIRE
Viridian Announces Inducement Grants Under Nasdaq Rule 5635(c)(4)
05 Nov 2024 //
BUSINESSWIRE
X4 Pharma Announces Inducement Grants Under Nasdaq LR 5635(c)(4)
01 Nov 2024 //
GLOBENEWSWIRE
BPGbio Receives FDA Rare Disease Designation for EB Treatment
31 Oct 2024 //
BUSINESSWIRE
Top 10 Rare Disease Pharma Sees 10.4% Therapy Initiation Increase
31 Oct 2024 //
BUSINESSWIRE
Quoin Pharmaceuticals to Report Q3 2024 Results on Nov 7
31 Oct 2024 //
GLOBENEWSWIRE
Revive Therapeutics Provides Bucillamine Nerve Agent Study Update
31 Oct 2024 //
GLOBENEWSWIRE
Agios Reports Business Highlights and Q3 2024 Financial Results
31 Oct 2024 //
GLOBENEWSWIRE
n-Lorem Foundation Treats 15 Nano-rare Patients, More Expected
30 Oct 2024 //
BUSINESSWIRE
Electra Gets FDA Orphan Drug Designation for ELA026 in HLH
30 Oct 2024 //
BUSINESSWIRE
Novartis, Apellis roll out rival datasets in rare kidney disease
28 Oct 2024 //
FIERCE PHARMA
Sparrow Pharma Completes Ph 2 Trial for Cushing’s Syndrome
28 Oct 2024 //
BUSINESSWIRE
Chiesi Reports Phase 3 Results of ELFABRIO® in Fabry Disease
28 Oct 2024 //
PR NEWSWIRE
Bora to Expand Rare Disease Portfolio with Acquisition of Pyros
26 Oct 2024 //
PRESS RELEASE
Modalis Receives Orphan Drug Designation for MDL-101 in CMD
25 Oct 2024 //
BUSINESSWIRE
AZ licenses discarded rare disease drug to Monopar Therapeutics
25 Oct 2024 //
FIERCE BIOTECH
KIND Receives Orphan Drug Designation for AND017 in SCD
25 Oct 2024 //
PR NEWSWIRE
Soligenix, Inc. to Present at the 2024 ThinkEquity Conference
24 Oct 2024 //
GLOBENEWSWIRE
FDA Grants Rare Pediatric Disease Designation To Omeros’ Zaltenibart
24 Oct 2024 //
BUSINESSWIRE
Marinus Pharmaceuticals Announces Results From TrustTSC Trial
24 Oct 2024 //
BUSINESSWIRE
FDA declines to approve Camurus` rare hormone disorder drug
23 Oct 2024 //
REUTERS
FDA Awards Orphan Drug Designation For Brain Cancer Drug By Franklin
23 Oct 2024 //
PR NEWSWIRE
Savara backs talk show episode to boost rare disease awareness
22 Oct 2024 //
FIERCE PHARMA
AscellaHealth Releases Q3 2024 Specialty Pipeline Digest™
22 Oct 2024 //
GLOBENEWSWIRE
Quoin Pharmaceuticals Expands International Trials For Netherton
22 Oct 2024 //
GLOBENEWSWIRE
Savara`s aPAP Highlighted On “The Balancing Act®” Airing
22 Oct 2024 //
BUSINESSWIRE
Nido Biosciences Receives Orphan Drug Designation For SMA
22 Oct 2024 //
BUSINESSWIRE
Remedy Pharmaceuticals Receives FDA Orphan Designation For CIRARA
22 Oct 2024 //
PR NEWSWIRE
Travere Therapeutics to Report Third Quarter 2024 Financial Results
21 Oct 2024 //
GLOBENEWSWIRE
Protara Granted FDA Fast Track For Choline Chloride Treatment
21 Oct 2024 //
GLOBENEWSWIRE
