Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.
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In 2015, Luke and Hayleigh Tate welcomed their daughter Maisy-Leigh into the world. However, following an emergency C-section, Maisy-Leigh was taken into critical care. The doctors noticed a number of…
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Man’s Charity Skydive Raises Coffin-Siris Syndrome Awareness
When things in her life felt out of control, Ellen Samuels turned to poetry. This wasn’t necessarily abnormal for Ellen. As an English professor at the University of Wisconsin-Madison, Ellen…
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Professor Writes Haikus Based on EDS Experience, Treatments
Have you ever heard of a biomarker? In short, a biomarker, or biological marker, is some sort of objective measure which can tell you something about your health or health…
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Research Discovers New Ulcerative Colitis Diagnostic Marker
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When Rachel Matthews birthed her twin boys, Aiden and Gavin, she was ecstatic. But just a day or two after Rachel had returned home with her boys, she received a…
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Mother of Twins Raises CAH Awareness
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There are three subsets of Von Willebrand disease (vWD), a genetic bleeding disorder. Type 3 is considered to be the most severe form of vWD, culminating in intense or severe…
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ICYMI: VONVENDI Approved for Von Willebrand Disease
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If you follow Major League Baseball (MLB), you’ve probably been inundated with news regarding the current lockout – and concerns over whether the season will begin on time. However, if…
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MLB Player Cedric Mullins Opens Up About Crohn’s Disease Diagnosis
How effective is UPLIZNA on treating attacks associated with neuromyelitis optica spectrum disorder (NMOSD)? According to a news release from biopharmaceutical company Horizon Therapeutics plc ("Horizon"), an analysis highlighted that…
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New Data Highlights UPLIZNA Efficacy for NMOSD
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Prior to February 2022, there were no approved treatments for those with cold agglutinin disease (CAD). However, that changed on February 4th, 2022, when the FDA announced that it had…
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Sutimlimab Now FDA-Approved for Cold Agglutinin Disease
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In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, prevent, or diagnose rare diseases or conditions. A “rare” condition is one affecting under…
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Ketamine Earns Orphan Drug Designation for Status Epilepticus
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BRUKINSA (zanubrutinib) is a prescription medication which can be used to treat Waldenstrom macroglobulinemia (WM), mantle cell lymphoma, and marginal cell lymphoma. So far, the treatment has been approved in…
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Switzerland Approves BRUKINSA for Waldenstrom Macroglobulinemia
Historically, both osteosarcoma and neuroblastoma have been difficult to treat. Therefore, new treatment options are needed for these cancers, which predominantly affect children and teenagers. According to Medical XPress, a…
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Antibody Combination Could Treat Neuroblastoma, Osteosarcoma
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Within 48 hours following her son Kylan's birth, Emily Bussenschutt had already visited three separate hospitals searching for a diagnosis. 9Honey explains that Kylan was born with epidermolysis bullosa…
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Mom Raises Epidermolysis Bullosa Awareness by Sharing Son’s Story
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Over the last few years, many researchers have been utilizing organoids as a pathway to learn more about diseases and spur drug development. Organoids may be helpful, moving forward, in…
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Chordoma Organoid Models Could Improve Drug Discovery
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In the past, many researchers have hypothesized a link between multiple sclerosis (MS) and different viruses or viral infections. While these hypotheses are varied in nature, there has rarely been…
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Research Highlights How EBV Can Trigger MS
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In a first-in-human, dose-escalation Phase 1 clinical trial, researchers are evaluating cosibelimab (CK-301) as a potential therapeutic option for those with metastatic cutaneous squamous cell carcinoma (cSCC). So far, shares Cancer…
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Cosibelimab Beneficial for cSCC, Research Shows
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In a recent news release, commercial-stage biopharmaceutical company HUTCHMED shared that its investigational therapy HMPL-523 had received Breakthrough Therapy designation from the National Medical Products Administration (NMPA) in China. HMPL-523…
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HMPL-523 Earns Breakthrough Therapy Designation in China for ITP
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Prior to February 2022, there were no approved disease-modifying therapies for those with pyruvate kinase (PK) deficiency. However, this has recently changed! According to a news release from biopharmaceutical company…
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PYRUKYND Approved in US for PK Deficiency
On February 16, 2022, biotechnology company Hillstream BioPharma Inc. (“Hillstream”) shared that its drug candidate HSB-1216 received Orphan Drug designation from the U.S. Food and Drug Administration. This particular designation…
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HSB-1216 Granted Orphan Drug Designation for Uveal Melanoma
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On February 15, 2022, biotechnology company Zealand Pharma A/S (“Zealand”) announced that its Phase 3 1703 clinical trial evaluating dasiglucagon for congenital hyperinsulinism (CHI) had reached full enrollment. Within this…
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Dasiglucagon Trial for Congenital Hyperinsulinism Reaches Full Enrollment
Researchers are consistently looking for ways to deepen their understanding of various conditions, which not only helps with drug development but with patient outcomes. According to Medical XPress, a research…
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Exploring IL-17A for Crohn’s Disease
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Prozac (fluoxetine) is used to treat a variety of mental health conditions including major depressive disorder (MDD), obsessive-compulsive disorder (OCD), bulimia, and premenstrual dysphoric disorder (PMDD), among others. You may…
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Could Prozac Treat Glioblastoma?
Stewart and Lorrie Altman
In Part 1 of our interview with Lorrie and Stewart Altman, we discussed the journey to the Late-onset Tay-Sachs disease (LOTS) diagnosis, how Lorrie and Stewart's outlook shaped their lives,…
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Interview: Living a Full and Filling Life with Late-Onset Tay-Sachs (LOTS) Pt. 2
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In a news release from early February 2022, clinical-stage biotechnology company Eureka Therapeutics, Inc. (“Eureka”) shared that two of its therapies – ET140203 and ECT204 – received Orphan Drug designation…
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ET140203, ECT204 Earn Orphan Drug Designation for Hepatocellular Carcinoma
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In late January 2022, biopharmaceutical company BridgeBio Pharma, Inc. ("BridgeBio") shared via news release that the first patient had been dosed in the Phase 1/2 ADventure clinical trial. Within this…
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ICYMI: First Patient Dosed in BBP-631 Trial for CAH
Stewart and Lorrie Altman
48 years of marriage, 2 children, 2 nephews, 7 grandchildren, and 3 great-nieces. It is clear that Lorrie and Stewart Altman, over their time together, have created a beautiful life.…
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Interview: Living a Full and Filling Life with Late-Onset Tay-Sachs (LOTS) Pt. 1