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» Rare Diseases
Rare Diseases
PDUFA VII is a step in the right direction for the rare disease community
RAPS.org
Tue, 09/7/21 - 10:51 pm
FDA
regulatory
PDUFA VII
Rare Diseases
Rare disease patients struggle for access to experimental drugs
Beckers Hospital Review
Sun, 07/18/21 - 09:36 pm
Rare Diseases
compassionate use guidelines
The "Rare Disease Company Coalition" Launches to Share the Unique Challenges and Promise of Rare Disease Therapy Development to Support Continued Progress and Patient Access
Yahoo/Businesswire
Thu, 05/13/21 - 12:04 pm
Rare Diseases
Rare Disease Company Coalition
drug development
drug discovery
Acceleron
Aeglea BioTherapeutics
Agios Pharmaceuticals
Alnylam
Harmony Biosciences
Orchard Therapeutics
Orphazyme
Sarepta Therapeutics
Taysha Gene Therapies
Ultragenyx
How Covid-19 is changing rare diseases research
MedCity News
Wed, 03/10/21 - 10:29 am
COVID-19
Rare Diseases
R&D
Pharmacquired: What a surprise deal does for AstraZeneca and Alexion
BioPharma Dive
Wed, 12/16/20 - 11:31 am
AstraZeneca
Alexion
M&A
Rare Diseases
AstraZeneca to buy Alexion for $39B
BioPharma Dive
Sat, 12/12/20 - 02:30 pm
AstraZeneca
Alexion
M&A
Rare Diseases
FDA to expedite development of two Sanofi rare disease drugs
Pharmaforum
Thu, 11/19/20 - 10:23 am
Sanofi
FDA
Rare Diseases
Pompe disease
Avalglucosidase alfa
Takeda drops $300M cash to kick off $1B RNAi alliance with Arrowhead, shooting for first-in-class rare disease drug
Endpoints
Thu, 10/8/20 - 10:59 am
Takeda
RNAi
Arrowhead Pharmaceuticals
Rare Diseases
FDA seeks input on rare disease clinical trials network
RAPS.org
Sat, 05/30/20 - 07:53 pm
FDA
Rare Diseases
clinical trials
With money flowing, Pliant Therapeutics heads to public well
Endpoints
Tue, 05/12/20 - 11:56 am
IPOs
Pliant Therapeutics
Rare Diseases
oncology
Takeda signs $882m rare disease alliance with Oxford biotech Evox
Pharmaforum
Fri, 03/27/20 - 10:34 am
Evox Therapeutics
Takeda
Rare Diseases
biotech
The biggest challenges facing rare disease pharma – and how the industry can tackle them
Pharmaforum
Sat, 02/29/20 - 07:17 pm
Rare Diseases
orphan drugs
drug development
FDA
drug pricing
Spark, Ipsen and more join advocacy groups, agencies in Rare Disease Day 2020 efforts
Fierce Pharma
Fri, 02/28/20 - 11:50 pm
Spark Therapeutics
Ipsen
Rare Diseases
Regeneron's bone disease drug delivers in phase 2, poised for FDA filing
Fierce Biotech
Thu, 01/9/20 - 10:55 am
Regeneron
Rare Diseases
FDA
clinical trials
garetosmab
3 Pharma & Biotech Pipeline Areas to Keep an Eye on in 2020
Yahoo/Zacks.com
Wed, 01/8/20 - 10:42 am
Alzheimer's disease
Medical Marijuana
Rare Diseases
Drugmakers rebranding around oncology, rare diseases could provide 2020 preview
Fierce Pharma
Thu, 12/19/19 - 11:32 pm
oncology
Rare Diseases
AstraZeneca
GSK
Alexion
Takeda
Soliris
Horizon looks to the future with massive San Francisco manufacturing, R&D plant
Fierce Pharma
Wed, 12/4/19 - 10:29 am
Horizon Therapeutics
eye health
R&D
drug manufacturing
Rare Diseases
San Francisco
Takeda sees cell, gene therapy in its future. Is it too late?
Biopharma Dive
Thu, 11/21/19 - 10:16 am
Takeda
gene therapy
Rare Diseases
Rare Disease Clinical Trials: FDA Awards $15M in Grants
RAPS.org
Tue, 10/8/19 - 09:06 pm
grants
clinical trials
FDA
Rare Diseases
Rare genetic disease company Homology raises $125M follow-on
BioCentury
Wed, 04/10/19 - 11:42 pm
Homology Med
Rare Diseases
gene therapies
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