Venclyxto® Plus Obinutuzumab Offers Previously Untreated CLL Patients Chemotherapy-Free Treatment
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Venclyxto® Plus Obinutuzumab Offers Previously Untreated CLL Patients Chemotherapy-Free Treatment

  A recent press release by the global biopharmaceutical research company, AbbVie, reported a positive opinion by CHMP granted to VENCLYXTO® combined with obinutuzumab to treat chronic lymphocytic leukemia (CLL). A positive opinion by…

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With Post-Brexit Move to Amsterdam Complete, EMA Renews Rare Disease Focus

The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over two…

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Patient Screening Completed for Clinical Trial of Zygel, a Treatment for Fragile X Syndrome
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Patient Screening Completed for Clinical Trial of Zygel, a Treatment for Fragile X Syndrome

Zynerba Pharmaceuticals has recently announced that they have finished the patient screening for their clinical trial of Zygel, a treatment for Fragile X syndrome. Zygel is a CBD gel that…

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Support “The Making of Justice” a Documentary About Seeking Treatment with a Rare Disease
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Support “The Making of Justice” a Documentary About Seeking Treatment with a Rare Disease

When Jennifer Payne was born in 1973, she was diagnosed with a rare disease: phenylketonuria (PKU). This genetic disorder is typically screened for at birth. This early screening process is…

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A Teenager’s Plan to Help her Friend with Diffuse Intrinsic Pontine Glioma Takes Off
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A Teenager’s Plan to Help her Friend with Diffuse Intrinsic Pontine Glioma Takes Off

According to a story from Essex Live, Lily Wythe is a fourteen year old girl who was recently diagnosed with a rare form of brain cancer called diffuse intrinsic pontine…

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Biogen to Relaunch its Alzheimer’s Drug with FDA Clearance in New Study
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Biogen to Relaunch its Alzheimer’s Drug with FDA Clearance in New Study

  According to an article in the Alzheimer’s publication Being Patient, participants who had been enrolled in the discontinued phase three trials of aducanumab will start receiving the drug as…

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Translarna Results in a Preserved Ability to Walk in Those with Duchenne Muscular Dystrophy
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Translarna Results in a Preserved Ability to Walk in Those with Duchenne Muscular Dystrophy

PTC Therapeutics is a pharmaceutical company that has been working with Translarna, a medication intended for the treatment of Duchenne muscular dystrophy (DMD). They analyzed this treatment and presented the…

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