Policy and
Public Affairs
Resources
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Articles
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Industry and trade associations
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Patient Advocacy Groups
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Podcast
Articles
Alabama will screen newborns for more rare diseases thanks to Foley teen, family and advocates
Publisher: AL.com
Articles
Biomarker breakthrough unleashes MPS therapies
Publisher: BioCentury
Articles
The rare pediatric disease voucher program creates new treatments. I have new data to prove it
Publisher: Stat
Articles
The FDA could help save my son from a rare disease
Publisher: WSJ
Articles
Too many treatable diseases go unnoticed. This could change that.
Publisher: Washington Post
Articles
"Bootcamp" helps rare disease advocates learn the ropes of drug development
Publisher: PharmaVoice
Articles
The FDA is at a crossroads on cell and gene therapies
Publisher: Stat
Articles
Aduhelm's accelerated approval offers a promising roadmap for rare neurological diseases
Publisher: Stat
Articles
Three misconceptions about the accelerated drug approval pathway
Publisher: Stat
Articles
My son’s time is running out due to a rare disease. The FDA needs to add more clinical trial flexibility
Publisher: Beyond Batten Disease Foundation
Articles
Gene therapy is in crisis. For nine hours, the field’s leading minds looked for a solution
Publisher: Stat
Articles
‘We’ve exhausted all avenues’: A small biotech may give up on its ultra-rare disease drug over frustration with FDA
Publisher: Stat
Articles
Rare-disease doctors support expanded newborn genomic screening, survey finds
Publisher: Stat
Articles
"No magic bullet": For drugmakers and the FDA, clinical trials on ultra-rare diseases pose thorny challenges
Publisher: Stat
Articles
My granddaughter’s devastating rare disease deserves attention, too
Publisher: Seattle Times
Articles
The FDA is failing children with a rare, neglected disease sometimes called "childhood dementia"
Publisher: Salon
Articles
Compassion or Cop-out?
Publisher: Real Clear Health
Articles
Does the orphan drug act need a tune-up?
Publisher: Pink Sheet
Articles
Accelerated approval in the US: It really is rare outside of oncology
Publisher: Pink sheet
Articles
Impasse at US FDA could mean stealth abandons Barth syndrome treatment
Publisher: Pink Citeline
Articles
Recommendations for the development of rare disease drugs using the accelerated approval pathway and for qualifying biomarkers as primary endpoints
Publisher: Orphanet journal of rare diseases
Articles
The future of medicine is unfolding before us. Are we nurturing It?
Publisher: NYT
Articles
The Barth syndrome foundation delivers petition to FDA advocating for a fair, equitable and appropriate review of the only potential treatment for Barth syndrome
Publisher: Not Too Rare to Care
Articles
Sheffield family plead for help after drug trial for boy, 11, is abruptly stopped
Publisher: The Guardian
Articles
Patient group pushes FDA to take action on drug
Publisher: Global Genes
Articles
Accelerated approval will be 'the norm' for gene therapies, FDA's Peter Marks says
Publisher: Endpoints
Articles
The transformation of drug development for the 21st century: Time for a change
Publisher: Molecular Genetics and Metabolism
Articles
It takes an average of 8 years for a rare disease patient to get diagnosed. Why is it so hard to get life-altering genetic testing in the U.S.?
Publisher: Fortune
Articles
Rare disease trials can't find enough patients. It's forcing the FDA to rethink its approach.
Publisher: Endpoints
Articles
The burden of rare diseases: An economic evaluation
Publisher: Chiesi
Articles
Accelerating a path to new treatments for rare neuronopathic MPS diseases
Publisher: Biospace
Articles
Tiny biotech says its troubles make case for ultra-rare FDA pathway
Publisher: BioCentury
Articles
Hopes, fears for orphan drug act
Publisher: BioCentury
Articles
A new paradigm for ultra-rare diseases
Publisher: BioCentury
Articles
Drug providing hope for Arizona toddler with rare genetic disorder
Publisher: Arizona's family
Articles
Ultrarare inflection point: stakeholders argue for biomarker-based approvals
Publisher: BioCentury
Articles
Feds' Biotech Enforcement Efforts Are Too Heavy-Handed
Publisher: Law360
Podcast
Rare Disease Spotlight: MPS & DMD
Publisher: BioCentury
Patient Advocacy Groups
Rare Ready Coalition
Patient Advocacy Groups
Global genes
Patient Advocacy Groups
NORD
Patient Advocacy Groups
Everylife foundation for rare diseases
Industry and trade associations
Institute for gene therapies
Industry and trade associations
ASGCT
Industry and trade associations
Alliance for regenerative medicine
Industry and trade associations
Rare disease company coalition
Industry and trade associations
Mass BIO
Industry and trade associations
California life sciences
Industry and trade associations