Rare Diseases

BioMarin Expands Enzyme Expertise With $270M Inozyme Buy
BioSpace
Fri, 05/16/25 - 11:45 am
Biomarin
M&A
Inozyme
INZ-701
Rare Diseases
FDA delays decision on Biohaven's rare disease drug filing, plans adcomm
Fierce Biotech
Thu, 05/15/25 - 11:02 am
Biohaven
FDA
troriluzole
adcomm
Rare Diseases
Alnylam keeps pushing the rare disease paradigm as RNAi promises even wider indications
Pharma Voice
Thu, 05/8/25 - 10:57 am
Alnylam
Rare Diseases
RNAi
R&D
FDA misses approval deadline for biotech’s rare disease drug
BioPharma Dive
Wed, 04/30/25 - 10:55 am
FDA
Stealth BioTherapeutics
drug approvals
Rare Diseases
elamipretide
Barth Syndrome
Makary Discusses Expedited Rare Disease Approvals Pathway, ‘Public Distrust’ in New Interview
BioSpace
Mon, 04/21/25 - 05:56 pm
FDA
Marty Makary
Rare Diseases
drug approvals
regulatory
Glycomine raises $115m to advance rare disease therapy into Phase IIb
Clinical Trials Arena
Thu, 04/17/25 - 11:05 am
Glycomine funding
GLM101
Rare Diseases
Norgine strengthens rare disease portfolio with acquisition of Theravia
Yahoo Finance
Wed, 04/16/25 - 11:18 am
M&A
Theravia
Norgine
Rare Diseases
Incentivizing rare disease R&D is getting tougher, but revamped policies could turn the tide
Pharma Voice
Tue, 02/25/25 - 10:47 am
R&D
Rare Diseases
artificial intelligence
2021 was the leading year in rare disease trials
Clinical Trials Arena
Mon, 02/3/25 - 11:49 am
clinical trials
Rare Diseases
FDA scraps adcomm for Applied's rare disease drug ahead of November decision
Fierce Biotech
Wed, 09/18/24 - 01:56 pm
Applied Therapeutics
govorestat
FDA
Rare Diseases
FDA pushed to be more open, collaborative to accelerate rare disease R&D
Fierce Biotech
Fri, 09/13/24 - 09:43 am
FDA
innovation
Rare Diseases
Roche tops pharma rivals for reputation among rare disease patient groups
Medical Marketing and Media
Wed, 08/21/24 - 11:39 am
Roche
reputation
Rare Diseases
Biomarin
Amgen
FDA launches innovation hub to spur rare disease R&D
Pharmaphorum
Fri, 07/19/24 - 11:37 am
FDA
Rare Diseases
R&D
Moderna’s mRNA Candidate Joins FDA’s Accelerator Program for Rare Diseases
BioSpace
Thu, 06/6/24 - 11:39 am
Moderna Therapeutics
mRNA-3705
FDA
Rare Diseases
START
Sanofi pays $80M to add Roche-rivaling Fulcrum drug to phase 3 rare disease pipeline
Fierce Biotech
Mon, 05/13/24 - 11:25 am
Sanofi
Fulcrum Therapeutics
Roche
GSK
Rare Diseases
clinical trials
losmapimod
Rare Disease Day Reminds Biopharma of Opportunities, Challenges
BioSpace
Fri, 03/1/24 - 11:43 am
Rare Diseases
Leveraging tRNA to Scale Genetic Medicines for Rare Diseases and Beyond
BioSpace
Mon, 12/11/23 - 09:56 am
tRNA
drug development
Rare Diseases
Inovio, with path to market opening up, drafts accelerated approval talking points ahead of FDA meeting
Fierce Biotech
Fri, 11/10/23 - 10:08 am
Inovio Pharmaceuticals
Rare Diseases
INO-3107
DNA
FDA
FDA Launches Pilot Program to Help Further Accelerate Development of Rare Disease Therapies
Applied Clinical Trials Online
Sun, 10/1/23 - 02:30 pm
FDA
Rare Diseases
clinical trials
drug development
DeepMind’s New AI Can Predict Genetic Diseases
Wired
Tue, 09/19/23 - 10:59 am
Google
DeepMind
AlphaMissense
artificial intelligence
DNA
R&D
Rare Diseases

Pages