Gene Therapy in Mouse Model Gives Insight into Possible SMA Treatments
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Gene Therapy in Mouse Model Gives Insight into Possible SMA Treatments

by Lauren Taylor from In The Cloud Copy Spinal muscular atrophy, or SMA, is a genetic disease that affects an individual’s central and peripheral nervous systems, as well as their…

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First Patient Treated in NKX101 Trial for AML, MDS

  Recently, biopharmaceutical company Nkarta announced the dosing of the first patient in its Phase 1 clinical trial. Within the trial, researchers will evaluate and analyze the company's investigational therapy,…

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$3.7M Grant Will Fund Malignant Infantile Osteopetrosis Research 
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$3.7M Grant Will Fund Malignant Infantile Osteopetrosis Research 

  In mid-November, 2020, Rocket Pharmaceuticals announced that the company received a $3.7M grant from the California Institute for Regenerative Medicine to study potential treatments for malignant infantile osteopetrosis. In…

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Cystic Fibrosis Patients May Now Be Able to Have Hearing Loss Screenings Done Via Tablets
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Cystic Fibrosis Patients May Now Be Able to Have Hearing Loss Screenings Done Via Tablets

by Danielle Bradshaw from In The Cloud Copy A recently performed study has demonstrated that it is possible to use tablets to screen patients that have chronic lung conditions -…

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New Phase 1 Study for Becker Muscular Dystrophy Treatment
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New Phase 1 Study for Becker Muscular Dystrophy Treatment

Edgewise Therapeutics has announced their plan to initiate a Phase 1 trial of EDG-5506, their small molecule therapy for Becker muscular dystrophy. It will take place at Worldwide Clinical Trials…

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Study Evaluates the Cost-Effectiveness of NASH and NAFLD Screening
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Study Evaluates the Cost-Effectiveness of NASH and NAFLD Screening

by Lauren Taylor from In The Cloud Copy Nonalcoholic fatty liver disease, or NAFLD, and nonalcoholic steatohepatitis, or NASH, are two conditions affecting the liver that plague millions of Americans…

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Two Clinical Trials for POMC and LEPR Deficiency Obesity Led to a Significant Reduction in Body Weight and Hunger

Rhythm Pharmaceuticals, based in Boston, MA, is a biopharmaceutical company focusing on the development of therapies to treat rare genetic diseases that cause obesity. Until now, there have not been…

Continue Reading Two Clinical Trials for POMC and LEPR Deficiency Obesity Led to a Significant Reduction in Body Weight and Hunger
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