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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
Oxford-Harrington Rare Disease Centre Announces 2025 Scholars Advancing Promising Treatments
November 21, 2025
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4 min read
Press Releases
Orbus Therapeutics Highlights Phase 3 STELLAR Clinical Study Results Showing Clinically Meaningful Benefits in Rare Brain Tumor at Society for Neuro-Oncology (SNO) Annual Meeting
November 21, 2025
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5 min read
Press Releases
Agios Announces Topline Results from RISE UP Phase 3 Trial of Mitapivat in Sickle Cell Disease
November 20, 2025
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17 min read
Press Releases
Medunik USA reaffirms support for Sickle Cell Disease community with uninterrupted supply and Patient-Centered Services
November 19, 2025
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10 min read
Press Releases
ReCode Therapeutics Initiates Enrollment of Phase 2 Clinical Trial of RCT2100 in Combination with Ivacaftor for the Treatment of Cystic Fibrosis
November 18, 2025
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3 min read
Press Releases
Profluent and RSRT Partner to Tackle Rett Syndrome with AI-Designed Custom Genomic Medicines
November 18, 2025
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3 min read
Press Releases
NORD Launches New RFP for Patient Registries
November 17, 2025
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3 min read
Press Releases
Neurogene Reports Third Quarter 2025 Financial Results and Highlights Recent Updates
November 14, 2025
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10 min read
Press Releases
Neurogene Reports Positive Interim Data in Pediatric Cohort from NGN-401 Gene Therapy Trial for Rett Syndrome
November 12, 2025
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9 min read
Press Releases
4DMT Reports Third Quarter 2025 Financial Results, Operational Highlights and Expected Upcoming Milestones
November 11, 2025
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10 min read
Press Releases
FDA Grants Orphan Drug Designation for Tinostamustine in Malignant Glioma
November 10, 2025
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5 min read
Press Releases
Soligenix Announces Recent Accomplishments and Third Quarter 2025 Financial Results
November 10, 2025
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9 min read
Press Releases
Neurogene Announces First Participant Dosed in Embolden™ Registrational Trial of NGN-401 Gene Therapy for Rett Syndrome
November 7, 2025
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7 min read
Press Releases
Taysha Gene Therapies Reports Third Quarter 2025 Financial Results and Provides Corporate Update
November 5, 2025
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14 min read
Press Releases
Medunik USA strengthens Pheburane® (sodium phenylbutyrate) coverage under Medicaid Programs
November 5, 2025
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9 min read
Press Releases
Sionna Therapeutics to Participate in Upcoming November 2025 Investor Conferences
November 5, 2025
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2 min read
Press Releases
Protara Therapeutics Named a 2026 Best Places to Work Winner by BioSpace
November 4, 2025
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2 min read
Press Releases
AMGEN REPORTS THIRD QUARTER 2025 FINANCIAL RESULTS
November 4, 2025
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46 min read
Press Releases
Tisento Therapeutics Announces Publication of MELAS Interview Study, Incorporating Patients’ Perspectives to Elucidate Symptoms and Impacts of Rare Mitochondrial Disease
November 4, 2025
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5 min read
Press Releases
ORYZON Announces First‑Patient‑In (FPI) in RESTORE Phase Ib Trial of Iadademstat in Sickle Cell Disease
November 4, 2025
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8 min read
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