Sanfilippo syndrome
Mar 19, 2025
Mucopolysaccharidosis: How the Different Types Affect Patients and Treatment Approaches?
Mucopolysaccharidosis (MPS) is a group of rare genetic disorders affecting roughly 1 in 25,000 births, yet its impact is profound. Caused by enzyme deficiencies, MPS leads to the buildup of complex sugars in cells, resulting in progressive damage to the heart, bones, brain, and other organs. Symptoms can vary widel...
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Feb 25, 2025
CTEXLI Approved for Cerebrotendinous Xanthomatosis; SIGX1094 Wins Fast Track for Diffuse Gastric Cancer; Bavarian Nordic’s Chikungunya Vaccine Cleared for Ages 12+; UX111 Gene Therapy Gains Priority Review for Sanfilippo Syndrome; SUBLOCADE Label Update Approved
FDA Approves CTEXLI, the First-Ever Treatment for Cerebrotendinous Xanthomatosis, a Rare Lipid Storage Disorder Mirum Pharmaceuticals, Inc. announced that the FDA has approved CTEXLI (chenodiol) tablets as the first and only treatment for cerebrotendinous xanthomatosis (CTX), a rare, progressive, and debilitatin...
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Jan 23, 2024
BMS, and Exelixis’s Opdivo + CABOMETYX in First-Line Advanced Renal Cell Carcinoma; AIRSUPRA Now Available as the First and Only FDA-approved Anti-inflammatory Rescue Option for Asthma; AstraZeneca’s Voydeya Receives First-ever Regulatory Approval; EMA Grants ODD to GC Biopharma’s Sanfilippo Syndrome (Type A) Treatment; FDA Approves NRx Pharma’s IND Application of NRX-101; FDA Fast Track Designation to Kyverna’s KYV-101
Opdivo in Combination with CABOMETYX Demonstrates Long-Term Survival Benefits After Four Years of Follow-Up in the CheckMate -9ER Trial in First-Line Advanced Renal Cell Carcinoma Bristol Myers Squibb and Exelixis, Inc. have released the four-year follow-up findings from the CheckMate -9ER trial, which investiga...
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Nov 04, 2019
The growing size of Sanfilippo syndrome Market: Drivers and Barriers
Sanfilippo syndrome, also known as mucopolysaccharidosis type III (MPS III), is a rare genetic disorder that affects the Central Nervous system. It is a result of a mutation in genes and can be of four types (A, B, C, and D). In all the four cases, the body fails to clear up the toxic sugars such as mucopolysacchar...
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