Pompe Disease

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Latest Pharma News and Updates for BeiGene, Seagen, Alnylam, Roche, Astellas
BeiGene’s Brukinsa Approval; FDA Approval to Seagen’s TUKYSA; NICE Recommends Alnylam’s Amvuttra; FDA Approves Brenzavvy for Type 2 Diabetes; Roche’s Tecentriq to be Filed for Early-stage Liver Cancer; FDA Lifts Hold on Astellas’ Pompe Gene Therapy

FDA Approves BeiGene’s Brukinsa for CLL/SLL BeiGene's Brukinsa (zanubrutinib) for chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL) has been approved by the US Food and Drug Administration. CLL is a common type of leukemia, accounting for approximately 25% of all new cases each year. SLL is...

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Pharma News and Updates for AbbVie, Ferring, Merck, CSL, Amicus, Takeda
Fourth FDA Approval for AbbVie’s Vraylar; FDA Approves Ferring’s Adstiladrin for NMIBC; Merck and Moderna’s mRNA Cancer Vaccine Trial; EMA Recommends the CSL’s Gene Therapy for Hemophilia B; CHMP Backs Amicus’ Pompe Disease Therapy; Takeda Announces the Phase 3 AURORA Study Result

AbbVie Secures Fourth FDA Approval for Vraylar AbbVie has received its fourth FDA approval for Vraylar, adding major depressive disorder (MDD) adjunctive therapy to a list that includes schizophrenia and manic and depressive episodes in bipolar disorder. According to AbbVie, the approval makes Vraylar (cariprazi...

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Top Most Expensive Drugs in the US Healthcare Market
Top 12 Most Expensive Drugs in the US Healthcare Market

Drug pricing is one of the hottest topics in the healthcare segment. Several arguments have been put forward by people, governments, healthcare companies, and other organizations in favor of and against expensive medicines. Some of the drugs are so expensive that they are nearly out of reach of the common people. H...

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Assessment of Key Products that Got FDA Approval in Second Half (H2) of 2021

Although COVID-19 continued to dominate headlines in 2021, pharmaceutical companies did not cease developing new treatments this year. The US Food and Drug Administration maintained a rapid rate of new drug approvals this year, all while managing the urgent examination of COVID-19 tests, treatments, and vaccines un...

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An Exploratory Analysis of the Rare and Inherited Pompe Disease
Pompe Disease: Causes, Symptoms, Diagnosis, Pathology, Current Marketed and Emerging Drugs in the Pipeline

Pompe disease is an inherited disorder caused by the buildup of a complex sugar called glycogen in the body's cells. The accumulation of glycogen in certain organs and tissues, especially muscles, impairs their ability to function normally. There are three types of Pompe disease, which differ in severity and the ag...

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Avrobio taps Magenta’s ADC; Eli Lilly ramps up; FDA postpones decision; CSL gears up to fight pandemic,

Avrobio taps ADC of Magenta for enhancing gene therapy conditioning Avrobio is working to make conditioning safer. The company is collaborating with Magenta Therapeutics to check antibody-drug conjugate. Under the agreement, the partners will evaluate Magenta’s lead conditioning program, MGTA-117, with at lea...

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Pompe disease Scenario in Eastern Europe
Pompe Disease Scenario in Eastern Europe

Pompe disease is an inherited rare disease, often fatal with a potential to damage heart, liver and skeletal muscles. Absence of an essential protein, Due to the mutation in a gene encoding GAA, there is an absence of a critical protein leading to a complex buildup of sugar, hence, damaging heart, liver and other o...

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Pompe Disease Emerging markets
Pompe Disease Emerging Markets

The global Pompe disease market will experience a boost during the study period of 2019-28. Pompe disease is a rare inheritable disorder with a combined Pompe disease prevalence of 1 in 40,000, affecting males and females equally. The Pompe disease therapeutic market revolves around pharmacological treatment (wh...

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Pompe disease Treatment Market
Pompe Disease Treatment Market

An orphan disease is any disease that affects only a small percentage of the population. There can be as many as 7,000 rare diseases known to affect human life. The total number of Americans living with a rare disease is estimated at between 25-30 million. When it comes to the EU, all together, rare disease patient...

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Pompe disease Market
Pompe Disease Market

Pompe disease, an inherited rare disease, is often fatal and incapacitates the heart and skeletal muscles. Absence of an essential protein due to the mutation in a gene encoding GAA, leads to a complex buildup of sugar, hence, damaging heart, liver and other organs. The mutated gene hampers the lysosomal-mediated d...

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