India's rare disease treatment funding fell to Rs 32.73 crore in 2025-26 from Rs 82.87 crore a year earlier, while several Centres of Excellence reported unspent balances. In Madhya Pradesh, concerns were also raised over the absence of dedicated rare disease centres in most state-run medical colleges.
Diagnosing rare diseases can take 3 to 15 years, while about 95% lack approved treatments. Families are advocating for regulatory changes to speed access to therapies as new digital tools aim to improve clinical trials.
Researchers discovered that adding three common amino acids to lipid nanoparticles dramatically improves mRNA and CRISPR gene editing delivery, boosting efficiency up to 20-fold and raising gene editing success rates from 25% to nearly 90%.
The global next generation drug conjugates market is projected to grow from $3.9 billion in 2026 to $10.9 billion by 2033 at 15.3% CAGR, while the radionuclide drug conjugate segment is forecast to reach $20.22 billion by 2032 at 9.6% CAGR.
The U.S. orphan drug market is projected to exceed $200 billion by 2031, driven by innovation in oncology and genetic disorders. More than 1,400 FDA-designated orphan drugs are in development or marketed, with over 800 currently in clinical trials.
A case report published in Oncotarget details a 38-year-old woman who developed acute lymphoblastic leukemia and lymphoblastic lymphoma shortly after receiving her second Pfizer-BioNTech mRNA vaccine dose, prompting calls for further investigation into potential immune-related mechanisms.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07493096 |
Intensive Multimodal Neurorehabilitation Targeting Neuroplasticity in Pediatric Neurodevelopmental and Chromosomal Disorders |
RECRUITING | |
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Whole Exome Sequencing and Whole Genome Sequencing for Nonimmune Fetal/Neonatal Hydrops |
RECRUITING | |
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COMPLETED | NA |
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Phase II, Randomized, Placebo-controlled Trial in Patients With Charcot-marie-tooth Disease Type 1A |
COMPLETED | PHASE2 |
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COMPLETED | PHASE2 |
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Face Anthropometric Pattern Recognition Technology for Computer Aided Diagnosis of Human Genetic Disorders. |
COMPLETED | |
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Allogeneic Bone Marrow Transplantation for the Treatment of Genetic Disorders of Erythropoiesis |
COMPLETED | PHASE2 |
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Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema |
COMPLETED | PHASE3 |
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Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema |
COMPLETED | PHASE2/PHASE3 |
| NCT00261053 |
Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema |
COMPLETED | PHASE2 |
