Brainstorm Shares the Results of its Phase 3 Study of NurOwn to Treat ALS
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Brainstorm Shares the Results of its Phase 3 Study of NurOwn to Treat ALS

  According to a report in BioPharma Dive, Brainstorm Therapeutics, a New York biotechnology company, said that while its NurOwn therapy for amyotrophic lateral sclerosis (ALS) appeared to have a…

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ICYMI: EC Approves OXLUMO for Patients with Primary Hyperoxaluria Type 1
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ICYMI: EC Approves OXLUMO for Patients with Primary Hyperoxaluria Type 1

  In late November, RNAi therapeutics company Alnylam Pharmaceuticals ("Alnylam") announced that its therapy OXLUMO (Lumasiran) received approval from the European Commission (EC) for both pediatric and adult patients with…

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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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New Discovery Allows Researchers to Study Mechanisms in Neurodegenerative Diseases from Beginning of life
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New Discovery Allows Researchers to Study Mechanisms in Neurodegenerative Diseases from Beginning of life

Dilated cardiomyopathy is a heart condition caused by a mutation in the RBM20 gene. This heart condition is unique because it can affect young adults. These individuals are at an…

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Possible Treatment for Pompe Disease to be Evaluated Under Priority Review
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Possible Treatment for Pompe Disease to be Evaluated Under Priority Review

According to a press release from the biopharmaceutical company Sanofi, the company's investigational treatment avalglucosidase alfa will have its Biologics License Application (BLA) evaluated by the US Food and Drug…

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Study Demonstrates Awareness and Research is Needed for Premenstrual Dysphoric Disorder

PMDD Premenstrual dysphoric disorder (PMDD) is a psychological condition which cyclically occurs with the menstruation cycle. Unfortunately, because the symptoms of this condition can mirror many mental health diagnoses, it…

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Study Shows Patients with Persistent GI Symptoms Should be Evaluated for Eosinophilic Gastritis/Duodenitis
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Study Shows Patients with Persistent GI Symptoms Should be Evaluated for Eosinophilic Gastritis/Duodenitis

A recent analysis has shown that eosinophilic gastritis and eosinophilic duodenitis are severely undiagnosed or misdiagnosed as another GI condition. Allakos Inc. has recently reported the results of this study,…

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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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Chronic Fatigue Syndrome Can Be Lonely, But There Are Ways to Cope
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Chronic Fatigue Syndrome Can Be Lonely, But There Are Ways to Cope

Research on myalgic encephalomyelitis, or chronic fatigue syndrome (ME/CFS), has developed in recent years; however, until now there has still been a gap in the literature regarding loneliness. This condition…

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Duchenne Muscular Dystrophy Trial has Gotten the Go Ahead from the FDA to Continue Dosing

Solid Biosciences has just reported that the FDA has finally lifted the hold they had placed on their Phase I/II trial for Duchenne muscular dystrophy (DMD). This trial is called…

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A New Treatment for BPDCN is on Track for EU Approval
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A New Treatment for BPDCN is on Track for EU Approval

According to a story from PR Newswire, the pharmaceutical and diagnostics company The Menarini Group recently announced that its drug tagraxofsup (marketed as Elzonris) has a received a positive opinion…

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Possible Treatment for Pompe Disease to be Evaluated Under Priority Review
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Possible Treatment for Pompe Disease to be Evaluated Under Priority Review

According to a press release from the biopharmaceutical company Sanofi, the Biologics License Application (BLA) for its drug avalglucosidase alfa has been accepted by the US Food and Drug Administration…

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Charcot-Marie-Tooth Disease Patients Report Declines During COVID-19 Lockdown
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Charcot-Marie-Tooth Disease Patients Report Declines During COVID-19 Lockdown

According to a story from Charcot-Marie-Tooth News, patients living with Charcot-Marie-Tooth disease have reported in a recent survey that their symptoms of pain and decline in mobility increased during the…

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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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Inclusive and Equally Effective Face Masks Can Make a Huge Difference for Those Living with Craniofacial Differences
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Inclusive and Equally Effective Face Masks Can Make a Huge Difference for Those Living with Craniofacial Differences

A recent article from the CCA has explained some considerations for mask types that you may not have thought about. Some of these are discussed below, but you can read…

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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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New Children’s Book for Kids with Craniofacial Differences is Published! “Simon and Patty Go To Camp”
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New Children’s Book for Kids with Craniofacial Differences is Published! “Simon and Patty Go To Camp”

Patricia Simon Patricia Simon was born with a cleft lip and palate. She is now a nurse, a public speaker, a patient advocate, and a writer. She has a great…

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Experimental Treatment for Sickle Cell Disease Earns Orphan Drug Designation in the EU
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Experimental Treatment for Sickle Cell Disease Earns Orphan Drug Designation in the EU

According to a story from Street Insider, the biopharmaceutical company Forma Therapeutics Holdings, Inc., recently announced that its investigational therapy candidate FT-4202 has earned Orphan Drug designation from the European…

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