Study: ANAVEX 3-71 as a Treatment for Neurodegenerative Diseases

Anavex Life Sciences Corporation has recently announced that they will conduct a clinical trial for their small molecule, ANAVEX®3-71. This therapy is intended to treat neurodegenerative and neurodevelopmental conditions, such…

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Sanfilippo Syndrome Treatment Receives Rare Pediatric Disease Designation
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Sanfilippo Syndrome Treatment Receives Rare Pediatric Disease Designation

Seelos Therapeutics has recently received the Rare Pediatric Disease Designation for their Sanfilippo syndrome treatment, trehalose. This designation is an incentive for companies to make therapies for conditions with an…

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European Commission Grants Conditional Approval for Spinal Muscular Atrophy Treatment

Zolgensma, a gene therapy for spinal muscular atrophy (SMA), has recently been granted conditional approval by the European Commission (EC). This is extremely exciting news for those living with SMA…

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Study: EDP-305 for the Treatment of Primary Biliary Cholangitis
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Study: EDP-305 for the Treatment of Primary Biliary Cholangitis

Enanta Pharmaceuticals has recently released the results from the second phase of its INTREPID trial, which studied EDP-305 as a treatment for primary biliary cholangitis (PBC). The results were positive,…

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New Therapy for ALS and Other Neurodegenerative Diseases
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New Therapy for ALS and Other Neurodegenerative Diseases

ProMIS Neurosciences is a biotechnology company that works specifically with antibody treatments, and they have recently developed intrabodies for the treatment of amyotrophic lateral sclerosis (ALS). This same therapy has…

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How this Sanfilippo Syndrome Patient Registry is Helping Patients
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How this Sanfilippo Syndrome Patient Registry is Helping Patients

The Connect MPS Patient Registry was created in 2016 to learn more about Sanfilippo syndrome and mucolipidoses (ML). 24 advocacy organizations are a part of this registry, all of which…

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New Genetic Test Can Predict Disease Progression of Idiopathic Pulmonary Fibrosis
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New Genetic Test Can Predict Disease Progression of Idiopathic Pulmonary Fibrosis

Predicting the disease progression of idiopathic pulmonary fibrosis (IPF) is extremely important, as this condition progresses differently depending on the affected individual. Knowing if one has a poor outcome while…

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