This Girl’s Diffuse Intrinsic Pontine Glioma Tumor is Gone, But No One Knows Why

According to a story from CBS News, Roxli Doss, an eleven year old girl, was diagnosed with a rare and dangerous brain tumor called diffuse intrinsic pontine glioma. It is…

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ICYMI: Creator of “SpongeBob SquarePants” Dies After Being Diagnosed With Amyotrophic Lateral Sclerosis
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ICYMI: Creator of “SpongeBob SquarePants” Dies After Being Diagnosed With Amyotrophic Lateral Sclerosis

According to a story from Buzzfeed News, Stephen Hillenburg, the creator of the smash hit children's cartoon SpongeBob SquarePants has passed away at age 57. Last year, Hillenburg had revealed…

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UK Cystic Fibrosis Patients are Protesting to get a Vital Drug Covered on the NHS
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UK Cystic Fibrosis Patients are Protesting to get a Vital Drug Covered on the NHS

Cystic fibrosis patients in the UK are planning a protest on November 19th, 2018 with the goal of getting Orkambi, an important cystic fibrosis drug, covered on the NHS. This…

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Want to Learn More About Clinical Trials? Don’t Miss This Free Webinar
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Want to Learn More About Clinical Trials? Don’t Miss This Free Webinar

The clinical research process is a thorough and time consuming method in which new drugs, therapies, and medical devices are rigorously tested before being made available to the public. Through…

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Data Looks Good For Synlogic’s Experimental Phenylketonuria Drug, And If You’re a Patient, You Can Get Involved
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Data Looks Good For Synlogic’s Experimental Phenylketonuria Drug, And If You’re a Patient, You Can Get Involved

The biotechnology company Synlogic announced positive data from its most recent Phase 1/2a clinical trial of its experimental product SYNB1618. This trial did not test the therapeutic action of the…

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Cystic Fibrosis Drug ORKAMBI Now Approved for Patients Age 2-5 by the FDA
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Cystic Fibrosis Drug ORKAMBI Now Approved for Patients Age 2-5 by the FDA

According to a story from BioSpace, an announcement from the biotechnology company Vertex Pharmaceuticals revealed that the U.S. Food and Drug Administration has approved an expanded indication for ORKAMBI, the…

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PatientWorthy is Offering Two Tickets to the First Annual Alagille Syndrome Alliance Gala
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PatientWorthy is Offering Two Tickets to the First Annual Alagille Syndrome Alliance Gala

PatientWorthy will be offering a pair of tickets to the Alagille Syndrome Alliance's (ALGSA) First Annual Gala. The tickets will be available for patients or caregivers in the San Diego…

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Express Scripts Makes Diagnosis Easier with Second Opinion
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Express Scripts Makes Diagnosis Easier with Second Opinion

According to PRNewswire, Express Scripts announced a new program to benefit people living with rare diseases on May 31st. The program, called Second Opinion is part of the company’s larger…

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Virginia Expands Medicaid Access to 400,000 Citizens, But What Does it Mean for Rare Patients?
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Virginia Expands Medicaid Access to 400,000 Citizens, But What Does it Mean for Rare Patients?

After years of resistance from the previously dominant Republican party, Virginia lawmakers were successfully able to pass a measure expanding Medicaid to nearly 400,000 more Virginians, according to the Washington…

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#MoveInMay – Check Out Rare Disease Work Out Tips on National Physical Education and Sport Week
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#MoveInMay – Check Out Rare Disease Work Out Tips on National Physical Education and Sport Week

Let's get moving in May! First off, for all you pop culture/Internet meme lovers, it's that time of year... May means we're on the eve of summertime, knee-deep in springtime,…

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New Drug Gains Approval For Treating X-Linked Hypophosphatemia in the U.S.
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New Drug Gains Approval For Treating X-Linked Hypophosphatemia in the U.S.

According to a story from globenewswire.com, the pharmaceutical companies Ultragenyx and Kyowa Kirin recently issued an announcement that the U.S. FDA has granted approval to their new treatment Crysvita (burosumab-twza).…

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The FDA Just Approved a New Maintenance Therapy for Chronic Inflammatory Demyelinating Polyneuropathy
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The FDA Just Approved a New Maintenance Therapy for Chronic Inflammatory Demyelinating Polyneuropathy

According to a story from raredr.com, The U.S. Food and Drug Administration (FDA) has recently approved Hizentra as a maintenance therapy for patients with chronic inflammatory demyelinating polyneuropathy. Hizentra is…

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Celebrate a Holiday your Kidney will appreciate: World Kidney Month!
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Celebrate a Holiday your Kidney will appreciate: World Kidney Month!

Last week was World Kidney Day! And in fact, the whole month of March is kidney month - so there's no excuse to brush up on some key kidney facts…

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