Experimental Non-Steroid Treatment Displays Potential for Congenital Adrenal Hyperplasia
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Experimental Non-Steroid Treatment Displays Potential for Congenital Adrenal Hyperplasia

According to a story from Healio, the results from a recent phase 2A clinical trial have demonstrated the effectiveness of an experimental drug in treating congenital adrenal hyperplasia (CAH). The…

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A Girl Diagnosed with Batten Disease got Treated with a Drug Developed Specifically for Her
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A Girl Diagnosed with Batten Disease got Treated with a Drug Developed Specifically for Her

According to a story from Technology Review, the story of Mila Makovec shows the potential impact that personalized medicine could have on patients while also revealing some of its limitations.…

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Phase 3 Clinical Trial for Amyotrophic Lateral Sclerosis Has Completed Enrollment
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Phase 3 Clinical Trial for Amyotrophic Lateral Sclerosis Has Completed Enrollment

BrainStorm Cell Therapeutics has just announced that they have completed enrollment in their Phase 3 clinical trial for amyotrophic lateral sclerosis (ALS). Enrollment was initiated way back in October of…

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Progressive Fibrosing Interstitial Lung Disease Drug Candidate Ofev Granted Breakthrough Therapy Status
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Progressive Fibrosing Interstitial Lung Disease Drug Candidate Ofev Granted Breakthrough Therapy Status

According to a publication from Scleroderma News, the American Food and Drug Administration (FDA) recently granted Breakthrough Therapy status to Ofev (generic name nintedanib), as a treatment for progressive fibrosing…

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Data Indicates That Gene Therapy for Spinal Muscular Atrophy Could be Useful for Older Patients
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Data Indicates That Gene Therapy for Spinal Muscular Atrophy Could be Useful for Older Patients

According to a story from MedCity News, interim data from a phase 1/2 clinical trial testing Zolgensma, a gene therapy for spinal muscular atrophy, in patients between the ages of…

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Two Nonprofits Award $200,000 Grant for Development of Duchenne Muscular Dystrophy Patient Reported Outcomes
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Two Nonprofits Award $200,000 Grant for Development of Duchenne Muscular Dystrophy Patient Reported Outcomes

According to a story from PR Newswire, the nonprofit groups Duchenne UK and Parent Project Muscular Dystrophy (PPMD) have jointly awarded grant funding to the tune of $200,000 to Dr.…

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Phase 1 Study of Experimental Systemic Sclerosis Treatment Bermekimab Begins
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Phase 1 Study of Experimental Systemic Sclerosis Treatment Bermekimab Begins

According to a recent press release from XBiotech, a Texas-based biotechnology company, patient dosing is now underway in a phase 1 clinical study of the Company's investigational systemic sclerosis treatment, bermekimab.…

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Grant to Fund Critical International Amyotrophic Lateral Sclerosis Research
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Grant to Fund Critical International Amyotrophic Lateral Sclerosis Research

According to a story from med.miami.edu, an international research consortium known as Clinical Research in ALS and Related Disorders for Therapeutic Development (CReATe) has just been awarded a renewal grant from…

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Personalized Medicine: A Drug Tailored for a Six Year Old With Batten Disease May Serve as a Template for Similar Rare Disorders

  Mila Makovec began her life as a normal, healthy infant, and according to a recent article in Science Magazine, she continued along this path until reaching the age of…

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