Cambridge Rare Disease Network’s RAREfest20

The Cambridge Rare Disease Network presents RARE Fest 2020 Engage.Educate.Empower. November 28, 2020 In light of the COVID-19 pandemic, RAREfest20 will be celebrated as a live virtual festival that will…

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RNA Interference and the Treatment of Rare Disease
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RNA Interference and the Treatment of Rare Disease

On November 19, 2020, Research!America hosted an informational webinar titled "From Nobel Winning Science to Next Generation Treatment: Tracing the Path of a Rare Disease Breakthrough using RNAi." This webinar focused…

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The Autoimmune Registry Releases Comprehensive List of Autoimmune Diseases
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The Autoimmune Registry Releases Comprehensive List of Autoimmune Diseases

According to a story from PR Newswire, The Autoimmune Registry, Inc. (ARI) has recently released its first ever autoimmune diseases list. This list includes a total of over 150 distinct…

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Navigating Rare Disease Clinical Trials in a Global Pandemic Means Putting Patients First

Navigating a Rare Disease in a Pandemic The COVID-19 pandemic has affected everyone, but those already living with a rare disease have unique challenges. With a compromised immune system, they…

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FDA Approves of Status Epilepticus Treatment, SESQUIENT
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FDA Approves of Status Epilepticus Treatment, SESQUIENT

Sedor Pharmaceuticals, a partner of Ligand Pharmaceuticals, has recently received FDA approval for SESQUIENT, its status epilepticus treatment. It has been cleared for the treatment of both adult and pediatric…

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New Combination for HRD-Positive Ovarian Cancer Gets EU Approval
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New Combination for HRD-Positive Ovarian Cancer Gets EU Approval

According to a story from Cancer Network, the European Commission has recently approved a two-part combination treatment for advanced, homologous recombination deficiency (HRD)-positive ovarian cancer. This first-line maintenance treatment consists…

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CCA Gives Tips for Transitioning into Adulthood with a Craniofacial Difference
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CCA Gives Tips for Transitioning into Adulthood with a Craniofacial Difference

Children's Craniofacial Association (CCA) has posted tips for transitioning to adulthood for those with craniofacial differences. We've summarized some of their recommendations, but you can read the full article here.…

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This Drug Could Boost Duchenne Muscular Dystrophy Treatment
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This Drug Could Boost Duchenne Muscular Dystrophy Treatment

According to a story from Newswise, trial results released in the journal PLOS Medicine suggest that the drug vamorolone could provide an enhancement in the effectiveness of corticosteroid therapy in patients…

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CymaBay Therapeutics Announces Results of Global Phase 3 ENHANCE Study for Primary Biliary Cholangitis

CymaBay Therapeutics, a biopharmaceutical company based in Newark, CA, develops and provides access to novel therapies with a recent focus on primary biliary cholangitis (PBC). The company is currently developing…

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AMO-02, a Congenital Myotonic Dystrophy Treatment, Granted Rare Pediatric Disease Designation
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AMO-02, a Congenital Myotonic Dystrophy Treatment, Granted Rare Pediatric Disease Designation

The FDA has recently granted the Rare Pediatric Disease (RPD) designation to AMO-02, a treatment for congenital myotonic dystrophy. As there is currently an unmet medical need for this condition,…

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Year Two of This Alport Syndrome Trial Concludes with Encouraging Findings
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Year Two of This Alport Syndrome Trial Concludes with Encouraging Findings

According to a story from GlobeNewswire, the biopharmaceutical company Reata Pharmaceuticals, Inc., recently announced that year two of its phase 3 clinical trial has been completed. This trial is testing…

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Study Identifies Clinical Characteristics of Chronic Nonbacterial Osteomyelitis
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Study Identifies Clinical Characteristics of Chronic Nonbacterial Osteomyelitis

According to a story from EurekAlert! a recent study presented at the annual meeting of the American College of Rheumatology describes some of the key clinical feature of chronic nonbacterial…

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A Combative Combination: Fighting Osteomyelitis with Stem Cells and Antibiotics
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A Combative Combination: Fighting Osteomyelitis with Stem Cells and Antibiotics

by Danielle Bradshaw from In The Cloud Copy Bone grafts, although very helpful, can result in hard to treat infections and typically result in the patient needing to be put…

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Presentation: Tyvaso as a Treatment for Interstitial Lung Disease
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Presentation: Tyvaso as a Treatment for Interstitial Lung Disease

United Therapeutics Corporation has recently presented data from the Phase 3 INCREASE trial, which evaluated Tyvaso as a treatment for pulmonary hypertension associated with interstitial lung disease. Results were presented…

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Hereditary Angioedema Patients in the UK Get Early Access to New Treatment
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Hereditary Angioedema Patients in the UK Get Early Access to New Treatment

According to a story from GlobeNewswire, the company BioCryst Pharmaceuticals, Inc., recently announced that its drug berotralstat (marketed as Orladeyo) will be available in the UK under the Early Access…

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