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Rare diseases

A patients story who suffered cystic fibrosis.
Drug Development
Cystic Fibrosis: One Patient’s Story, Current Treatments and Exciting New Therapies on the Horizon
“The recent progress of cystic fibrosis drugs has been amazing and, in my case, miraculous,” said Kelly Peters, who lives with cystic fibrosis. “The new drugs are not a cure, but they feel pretty close.”
May 10, 2021
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13 min read
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Chelsea Weidman Burke, M.S.
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PRESS RELEASES
Press Releases
Chiesi Global Rare Diseases Announces European Commission Approval of LOJUXTA® (lomitapide) ▼ Capsules for Paediatric Use in Homozygous Familial Hypercholesterolaemia (HoFH)
June 5, 2026
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5 min read
Press Releases
Quoin Pharmaceuticals Announces Japan’s MHLW Grants Orphan Drug Designation for QRX003 in Netherton Syndrome
June 4, 2026
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6 min read
Press Releases
Mirum Pharmaceuticals and Incyte Announce Pivotal Late-Breaking Results for Zilurgisertib in Fibrodysplasia Ossificans Progressiva Accepted for Presentation at ENDO 2026
June 4, 2026
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8 min read
Press Releases
Alex “A-Rod” Rodriguez to Deliver Special Remarks at The Foundation for Sickle Cell Disease Research’s 20th Annual Sickle Cell Disease Research and Educational Symposium and 49th National Sickle Cell Disease Scientific Meeting
June 4, 2026
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3 min read
Press Releases
Regeneron to Highlight Progress Across Its Metabolic Disease, Ophthalmology and Rare Disease Pipelines at ADA and ENDO
June 4, 2026
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10 min read
Press Releases
AMGEN TO PRESENT NEW DATA ACROSS RARE AUTOIMMUNE AND INFLAMMATORY DISEASES AT EULAR 2026
June 3, 2026
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30 min read
Press Releases
Ryan Clark to Deliver Warrior Keynote at The Foundation for Sickle Cell Disease Research’s 20th Annual Sickle Cell Disease Research and Educational Symposium and 49th National Sickle Cell Disease Scientific Meeting
June 3, 2026
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3 min read
Press Releases
IDefine and UT Southwestern Announce Research Collaboration to Advance Gene Therapy for Rare Disease Kleefstra Syndrome
June 3, 2026
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2 min read
Press Releases
Medicus Pharma Advances SkinJect® into Registrational Development for Gorlin Syndrome
June 3, 2026
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11 min read
Press Releases
Travere Therapeutics Enters Into Exclusive Licensing Agreement with Everest Medicines for Civorebrutinib a Potential Best-in-Class BTK Inhibitor for Rare Kidney Diseases
June 2, 2026
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11 min read
Press Releases
High-Precision Base-Editing Therapy Demonstrates Durable VOC-Free Efficacy and Favorable Safety in Sickle Cell Disease
June 2, 2026
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4 min read
Press Releases
Fulcrum Therapeutics Announces Discontinuation of Pociredir Program in Sickle Cell Disease and Initiation of Strategic Review
June 2, 2026
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7 min read
Press Releases
Mirum Pharmaceuticals Announces New Data from Rare Liver Disease Programs Presented at the EASL International Liver Congress 2026
June 1, 2026
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12 min read
Press Releases
Children’s Hospital Colorado Leads Nation in Treatment for Cystic Fibrosis Before Birth
May 27, 2026
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4 min read
Press Releases
Sionna Therapeutics to Participate in Upcoming June 2026 Investor Conferences
May 27, 2026
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1 min read
Press Releases
Vanda Pharmaceuticals Announces Orphan Drug Designation in Japan for Imsidolimab in Generalized Pustular Psoriasis
May 27, 2026
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4 min read
Press Releases
Suitability of Vaccine Platform for Bundibugyo Virus, Cause of the Ebola Outbreak in Congo
May 26, 2026
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13 min read
Press Releases
Avanzanite Bioscience’s Partner Agios Announces PYRUKYND® (mitapivat) Approval in the European Union for Adults with Thalassaemia
May 24, 2026
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5 min read
Press Releases
Agios’ PYRUKYND® (mitapivat) Approved in the European Union for Adults with Thalassemia
May 24, 2026
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15 min read
Press Releases
AstraZeneca to showcase Phase III data in liver, breast and bladder cancers and potential first-in-class rare disease therapy at ASCO 2026
May 22, 2026
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19 min read
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