CIRM Awards $12 Million Grant for Clinical Study of Gene Therapy for Cystinosis Patients
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CIRM Awards $12 Million Grant for Clinical Study of Gene Therapy for Cystinosis Patients

According to a press release from the California Institute for Regenerative Medicine (CIRM), the Institute's governing body has approved a grant of nearly $12 million to University of California, San…

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The IND Approval From The FDA, Experimental Acromegaly Drug Will Enter Phase 3 Trials
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The IND Approval From The FDA, Experimental Acromegaly Drug Will Enter Phase 3 Trials

According to a story from Markets Insider, the biopharmaceutical company Camurus has recently issued an announcement indicating that the US Food and Drug Administration (FDA) has accepted the company's Investigational…

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Accelerated Approval Granted to a Novel Drug Combination That Treats Patients with a Common Type of Non-Hodgkin Lymphoma

  Each year over eighteen thousand people in the United States are diagnosed with diffuse large B-cell lymphoma (DLBCL) a common type of non-Hodgkin lymphoma. A recent FDA news release…

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Don’t Miss VWD Connect Foundation’s First-Ever Type 3/Severe Von Willebrand Disease Patient Conference
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Don’t Miss VWD Connect Foundation’s First-Ever Type 3/Severe Von Willebrand Disease Patient Conference

Von Willebrand disease is the most common inherited blood clotting disorder that affects humans. The disorder can also be acquired. Although it is considered common, the majority of cases do…

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Scholarships are Available for the 2019 International Pain Summit Which Will be Held in LA this November

IPain The International Pain Foundation (IPain) is a nonprofit organization dedicated to supporting those with chronic pain due to neurological, musculoskeletal, inflammatory, degenerative, and emotional conditions. They aim to improve…

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The First Ever PFIC Network Family Conference will Bring Families Affected by the Disease Together This Week

Emily Ventura has never met in-person another person living with progressive familial intrahepatic cholestasis (PFIC), her daughter’s life-threatening ultra-rare genetic disease. That will change on June 21st when Emily and…

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Experimental Treatment for Hunter Syndrome Earns Rare Pediatric Disease Designation and Orphan Drug Designation
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Experimental Treatment for Hunter Syndrome Earns Rare Pediatric Disease Designation and Orphan Drug Designation

According to a story from globenewswire.com, the biopharmaceutical company Denali Therapeutics, Inc. has recently announced that its experimental product candidate DNL310, which is currently being developed as a treatment for…

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Critical Research Into Amyotrophic Lateral Sclerosis Therapies Gets Funding Boost
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Critical Research Into Amyotrophic Lateral Sclerosis Therapies Gets Funding Boost

According to a story from Alabama News Center, a scientist affiliated with Southern Research, Dr. Rita Cowell, has been hard at work researching new potential treatments for amyotrophic lateral sclerosis,…

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Presentations at the ATS Conference Demonstrate the Value of the Pulmonary Fibrosis Foundation’s Patient Registry

At the 2019 American Thoracic Society Conference this year multiple presentations demonstrated the importance of the Pulmonary Fibrosis Foundation's Patient Registry. About the Registry This registry was created by the Pulmonary…

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Companies Announce Plan to Offer Free Genetic Tests for Patients With Suspected WHIM Syndrome or Severe Congenital Neutropenia

According to a story from Business Wire, the biopharmaceutical company X4 Pharmaceuticals, Inc. and the medical genetics company Invitae Corporation have recently announced that they have come into a parternship…

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Issues with Quality Assurance of Generic Drugs and How We Can Improve the Process

What are Generic Drugs? Generic drugs are any medications "equivalent" to brand name pharmaceuticals. These drugs are much cheaper than the brand name medications and the United States Food and…

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