When the Latest Treatments are Out of Reach, Familial Hypercholesterolemia Patients Suffer
geralt / Pixabay

When the Latest Treatments are Out of Reach, Familial Hypercholesterolemia Patients Suffer

According to a story from FHM Pakistan, a recent study revealed the familial hypercholesterolemia patients are at a greater risk of stroke, heart attack, and other cardiovascular problems when they…

Continue Reading When the Latest Treatments are Out of Reach, Familial Hypercholesterolemia Patients Suffer
Biosimilar Drug for Ankylosing Spondylitis gets Approved by the FDA
stevepb / Pixabay

Biosimilar Drug for Ankylosing Spondylitis gets Approved by the FDA

According to a story from Ankylosing Spondylitis News, the drug Hadlima, which is a biosimilar to the drug Humira (adalimumab), was recently approved by the US Food and Drug Administration…

Continue Reading Biosimilar Drug for Ankylosing Spondylitis gets Approved by the FDA
Estela Lugo of the Hereditary Neuropathy Foundation Seeks to Raise Charcot-Marie-Tooth Disease Awareness
harpenz / Pixabay

Estela Lugo of the Hereditary Neuropathy Foundation Seeks to Raise Charcot-Marie-Tooth Disease Awareness

According to a story from Charcot-Marie-Tooth Disease News, Charcot-Marie-Tooth disease is actually somewhat common in the US compared to other rare diseases. In fact, it is estimated that around 150,000…

Continue Reading Estela Lugo of the Hereditary Neuropathy Foundation Seeks to Raise Charcot-Marie-Tooth Disease Awareness
FDA Grants Regenerative Medicine Advanced Therapy Designation to Experimental Wiskott-Aldrich Syndrome Drug
stevepb / Pixabay

FDA Grants Regenerative Medicine Advanced Therapy Designation to Experimental Wiskott-Aldrich Syndrome Drug

According to a press release from the British-American biopharmaceutical company Orchard Therapeutics, the American Food and Drug Administration (FDA) has granted OTL-103, the Company's experimental Wiskott-Aldrich Syndrome drug, Regenerative Medicine…

Continue Reading FDA Grants Regenerative Medicine Advanced Therapy Designation to Experimental Wiskott-Aldrich Syndrome Drug
Immunocore and Pulse Infoframe Lend Support to International Uveal Melanoma Patient Registry
Source: Pixabay

Immunocore and Pulse Infoframe Lend Support to International Uveal Melanoma Patient Registry

According to a press release from British biotechnology company Immunocore, the Company has partnered with Pulse Infoframe (a Canadian data analytics company) to support the development of an international database…

Continue Reading Immunocore and Pulse Infoframe Lend Support to International Uveal Melanoma Patient Registry
Possible Treatment for WHIM Syndrome Earns Orphan Drug Designation in the EU
Free-Photos / Pixabay

Possible Treatment for WHIM Syndrome Earns Orphan Drug Designation in the EU

According to a story from home.suddenlink.net, the biopharmaceutical company X4 Pharmaceuticals recently announced that the European Commission has granted Orphan Drug designation to the company's lead investigational drug candidate, mavorixafor.…

Continue Reading Possible Treatment for WHIM Syndrome Earns Orphan Drug Designation in the EU
Researchers Discover “Testicular Cancer-Associated Paraneoplastic Encephalitis,” an Autoimmune Disease
derneuemann / Pixabay

Researchers Discover “Testicular Cancer-Associated Paraneoplastic Encephalitis,” an Autoimmune Disease

According to a story from Cancer Health, a team of scientists affiliated with Mayo Clinic, UC San Francisco, and the Chan Zuckerburg Biohub have discovered a new rare, autoimmune disease…

Continue Reading Researchers Discover “Testicular Cancer-Associated Paraneoplastic Encephalitis,” an Autoimmune Disease
Researchers Continue Search for Role of Type-1 Interferon Pathway in Rheumatic Disease
mohamed_hassan / Pixabay

Researchers Continue Search for Role of Type-1 Interferon Pathway in Rheumatic Disease

According to a publication from the Rheumatologist, past studies have suggested that type 1 interferons may play a significant role in determining susceptibility to several classic connective tissue diseases. Although…

Continue Reading Researchers Continue Search for Role of Type-1 Interferon Pathway in Rheumatic Disease
Experimental Treatment for Neurofibromatosis Type 1 Earns Orphan Drug Designation in the EU
pixel2013 / Pixabay

Experimental Treatment for Neurofibromatosis Type 1 Earns Orphan Drug Designation in the EU

According to a story from BioPortfolio, the biopharmaceutical company SpringWorks Therapeutics, Inc., has recently announced that the European Commission has given the company's investigational drug candidate mirdametinib Orphan Drug designation.…

Continue Reading Experimental Treatment for Neurofibromatosis Type 1 Earns Orphan Drug Designation in the EU
Company Seeks to Treat Hunter Syndrome (MPS II) in China With New Drug Application
liaoyiye / Pixabay

Company Seeks to Treat Hunter Syndrome (MPS II) in China With New Drug Application

According to a story from BioSpace, the biopharmaceutical company CANBridge Pharmaceuticals announced that it has recently submitted a New Drug Application with China's National Medicinal Products Administration (NMPA) for its…

Continue Reading Company Seeks to Treat Hunter Syndrome (MPS II) in China With New Drug Application
Experimental Treatment for Primary Biliary Cholangitis Earns Orphan Drug Status in the US and EU
Aymanejed / Pixabay

Experimental Treatment for Primary Biliary Cholangitis Earns Orphan Drug Status in the US and EU

According to a story from Markets Insider, the biopharmaceutical company GENFIT recently announced that both the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) have awarded…

Continue Reading Experimental Treatment for Primary Biliary Cholangitis Earns Orphan Drug Status in the US and EU