May is GBS and CIDP Awareness Month: Spreading Rare Disease Awareness
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May is GBS and CIDP Awareness Month: Spreading Rare Disease Awareness

The month of May is recognized as Guillain-Barré syndrome (GBS) and chronic inflammatory demyelinating polyneuropathy (CIDP) awareness month, a time set aside to spread awareness about these rare conditions among…

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Community is Essential for Lambert-Eaton Myasthenia Syndrome Patients During the Pandemic

Quarantine during COVID-19 has been difficult for everyone. It's socially isolating, mentally draining, and physically limiting. We all find community different places. But no matter where we find it, it…

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Phase 3 Study for Primary Axillary Hyperhidrosis Has Demonstrated Positive Results

Brickell Biotech has just announced the findings from their Phase 3 study investigating the long-term efficacy and safety of sofpironium bromide gel for the treatment of primary axillary hyperhidrosis. Hyperhidrosis…

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Experimental Duchenne Muscular Dystrophy Treatment Earns Rare Pediatric Disease Designation
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Experimental Duchenne Muscular Dystrophy Treatment Earns Rare Pediatric Disease Designation

According to a story from apnews.com, the biopharmaceutical company FibroGen, Inc., has recently announced that its investigational treatment pamrevlumab has earned Rare Pediatric Disease designation from the US Food and…

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Ways to Support Those with Pediatric Growth Hormone Deficiency
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Ways to Support Those with Pediatric Growth Hormone Deficiency

Growth hormone deficiency (GHD) influences patients both physically and mentally. The condition means patients' bodies don't produce enough growth hormone. As a result, they don't develop the same way as…

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Study Illuminates Treatment Options for Different Pediatric Adrenocortical Carcinoma Stages

A study published in the Journal of Clinical Oncology has detailed the benefits of different forms of treatment for pediatric patients with adrenocortical carcinoma. These researchers particularly looked at what treatments…

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Phase 3 Trial for Pediatric Growth Hormone Deficiency in China Completed Enrollment On Time Despite COVID-19 Setback

VISEN Pharmaceuticals has recently announced that enrollment has been completed for their Phase 3 clinical trial examining the safety and efficacy of TransCon hGH compared to daily rhGH in patients…

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“Long COVID” Has Brought New Attention to Myalgic Encephalomyelitis
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“Long COVID” Has Brought New Attention to Myalgic Encephalomyelitis

According to a story from PR Newswire, Solve M.E. is gathering virtually on Capitol Hill for its 5th Annual Advocacy Week, starting April 19th. A record number of people are…

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April 23 is International FOP Awareness Day: Spreading Rare Disease Awareness
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April 23 is International FOP Awareness Day: Spreading Rare Disease Awareness

The International Fibrodysplasia Ossificans Progressiva Association (IFOPA) is gearing up to recognize International FOP Awareness Day this year on April 23, 2021. The event is meant to help spread awareness…

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Results from Phase 1 HATTR Amyloidosis Trial Presented at Recent Meeting
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Results from Phase 1 HATTR Amyloidosis Trial Presented at Recent Meeting

According to a story from globenewswire.com, the clinical company Prothena Corporation plc recently presented encouraging results from its phase 1 clinical trial. This trial was evaluating its investigational product candidate…

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