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Nov 26, 2024
Ractigen Therapeutics Receives FDA Orphan Drug Designation for RAG-21 to Treat ALS Ractigen Therapeutics has announced that the FDA has granted Orphan Drug Designation (ODD) to RAG-21, its novel siRNA therapy for treating amyotrophic lateral sclerosis (ALS). RAG-21 specifically targets FUS-ALS, one of the most a...
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Sep 24, 2024
The amyotrophic lateral sclerosis treatment landscape is stringent and includes multidisciplinary care, such as physical therapy, speech therapy, dietary counseling, heat or whirlpool therapy, and others. The approved drugs for amyotrophic lateral sclerosis treatment include RILUZOLE, RADICAVA, TIGLUTIK, EXSERVAN, ...
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Sep 24, 2024
DelveInsight forecasts that with the launch of 15+ emerging therapies alongside existing treatments, the ALS treatment market is anticipated to surpass USD 4 billion across the 7MM by 2034. Currently, there is no cure for ALS and no effective treatment to halt or reverse the progression of the disease. As per ou...
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Sep 24, 2024
Amyotrophic Lateral Sclerosis (ALS) or Lou Gehrig’s disease is a rare, progressive, and fatal neuromuscular disease characterized by degeneration of upper and lower motor neurons. While it is associated with a mortality rate of 50% within 30 months of Amyotrophic Lateral Sclerosis symptoms onset, there is still no ...
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Sep 24, 2024
Amyotrophic Lateral Sclerosis (ALS) is a devastating neurodegenerative disease characterized by the progressive degeneration of motor neurons, leading to muscle weakness, paralysis, and ultimately, death. Over the past decade, a growing understanding of the disease's underlying mechanisms has led to significant adv...
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Feb 27, 2024
Survodutide Phase II trial Shows Groundbreaking Results in Liver Disease due to MASH, with Significant Improvements in Fibrosis Boehringer Ingelheim has reported that in a Phase II trial, a significant proportion of adults treated with survodutide (BI 456906), up to 83.0%, showed a notable enhancement in metabol...
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May 02, 2023
FDA Grants Priority Review to Luspatercept for First-line Treatment of Anemia in Lower-risk MDS The FDA has granted priority review to a supplemental biologics license application (sBLA) seeking to expand the current indication of luspatercept-aamt (Reblozyl) to include treatment of anemia in patients with very ...
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Apr 04, 2023
HUTCHMED Completes Rolling Submission of NDA to FDA for Fruquintinib HUTCHMED (China) Limited announced the completion of the rolling submission of a New Drug Application ("NDA") to the United States Food and Drug Administration ("FDA") for fruquintinib, its highly selective and potent oral inhibitor of VEGFR-1,...
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Feb 28, 2023
Sage Therapeutics Receives EU Orphan Drug Designation for Huntington's Disease Treatment Candidate Sage Therapeutics declared the European Medicines Agency granted Orphan Drug Designation to SAGE-718 for the treatment of Huntington's disease. SAGE-718 is developing as a potential oral therapy for cognitive disor...
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Feb 07, 2023
Merck Wins Another FDA Approval for Blockbuster Keytruda Merck & Co arrived just two months after GSK celebrated a positive phase III result with its checkpoint inhibitor Jemperli as a first-line therapy for endometrial cancer. Keytruda (pembrolizumab) from Merck improved progression-free survival (PFS) vers...
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Roche’s HER2-Positive Breast Cancer Treatment Franchise
Aug 13, 2024
Transforming Multiple Myeloma Treatment: The Promise of Novel Drug Classes
Jul 24, 2024
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