Rare Disease
FDA Signals Shift Toward Bayesian Methods in Clinical Trials
As reported on MedCity News, the U.S. Food and Drug Administration (FDA) has issued draft guidance encouraging the thoughtful use of Bayesian statistical approaches in
Hereditary Cancer Risk Is Harder to Detect When Family History Crosses Borders
- June 18, 2026
FDA Grants Accelerated Approval to Teplizumab for Newly Diagnosed Pediatric Type 1 Diabetes
Alkermes’ Alixorexton Gains Orphan Drug Status in the US and EU
AstraZeneca’s Ravulizumab Receives FDA Priority Review for IgA Nephropathy
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THE LATEST FROM PATIENT WORTHY
Thyroid eye disease
Glycemic Monitoring Recommended for All Patients Receiving Teprotumumab in Thyroid Eye Disease
Emerging evidence, as reported by the American Academy of Ophthalmology, suggests that glycemic surveillance should be considered for all patients undergoing teprotumumab therapy for thyroid
Rare Disease
Aortic Valvotomy, A Widening of Heart Valves
When Seth Wharton was only one month old, doctors repaired his narrow heart valve. Then, five years later, he was referred to UAB Cardiovascular Institute
Dravet Syndrome
An Update from the Dravet Syndrome Foundation
Madrid, June 5th, 2026 The Dravet Syndrome Foundation received the Merck Innovation Talent Award for a scientific publication regarding a tool that assesses neuropsychiatric issues
Neurotrophic Keratopathy
TearSolutions Advances Lacripep® for Neurotrophic Keratitis with FDA Designations and Phase 2 Trial Launch
As reported on Business Wire, TearSolutions, a clinical-stage biotechnology company focused on ophthalmic therapies, has announced key regulatory and clinical progress for its investigational drug
Fabry Disease
Fabry International Network’s Young Adult Guidebook
Website: https://fabrynetwork.org/youngadults Instagram Young Adults: @fabryyoungadults Instagram FIN: @fabryintnetwork 11 June 2026 – FIN launches international Young Adults Guidebook created by and for young adults
Parkinson's Disease
Finding Balance: David and Charice’s Journey with Parkinson’s Disease
When David Williams noticed a slight tremor in his right hand, he convinced himself it was something manageable, perhaps a compressed nerve that would eventually
Rare Disease
FDA Clears New Sunscreen Ingredient After Two-Decade Gap
In a notable update for dermatologic and consumer health, the U.S. Food and Drug Administration (FDA) released a press statement disclosing their authorization of the
Cancer
FDA’s Real-Time Clinical Trial Model: Transforming Oncology Care While Raising New Challenges
As reported on Becker’s Hospital Review, the U.S. Food and Drug Administration’s launch of a real-time clinical trial model marks a notable shift in how
Cancer
Onchilles Pharma Initiates First-in-Human Trial of N17350 for Advanced Solid Tumors
As reported on Business Wire, Onchilles Pharma has announced the first patient dosing in a Phase 1/2 clinical trial evaluating its investigational therapy, N17350, in
Diabetes Type 2
Eli Lilly Advances Oral GLP‑1 With Strong Phase 3 Data in Type 2 Diabetes
As reported on BioSpace, Eli Lilly is moving closer to expanding the use of its oral GLP‑1 receptor agonist orforglipron, marketed as Foundayo, into type
Rare Disease
Omnix Medical Advances OMN6 Into Phase II Trial for Drug-Resistant Acinetobacter Infections
As reported on PharmaBiz, Omnix Medical has reached a key clinical milestone with the initiation of patient dosing in a phase II study of its
Pancreatic Cancer
Early Clinical Trial Suggests Promise for New Pancreatic Cancer Drug Combination
As reported on STAT News, a novel combination of investigational therapies from Tango Therapeutics and Revolution Medicines is generating cautious optimism after demonstrating notable tumor
Rare Disease
You Are More Than Your Diagnosis: A Message From a Mom With Multiple Myeloma
The questions I get asked the most are “How did it start?” “Did you have any symptoms?” “How did they find it?” For five years
HIV/AIDS
Merck and Gilead Post HIV Milestone Amid Setback in Lung Cancer Program
Merck and Gilead Sciences reported contrasting late-stage outcomes on Monday, pairing a promising advance in HIV treatment with the discontinuation of a high-profile lung cancer
Cancer
Stanford Medicine Achieves First Clinical Use of Compact Upright Proton Therapy
As reported on Business Wire, Stanford Medicine Cancer Center has reported a significant milestone in radiation oncology, completing the world’s first patient treatment using a
Rare Disease
“Hello, My Name Is”
Editor’s Note: This article was originally written and shared with us by Nick Leon of Naked Eye, and is shared here with gratitude. To see
Glioblastoma
MediciNova Advances Intellectual Property for Glioblastoma Combination Therapy
As reported by The Manila Times, MediciNova has announced that the U.S. Patent and Trademark Office has issued a Notice of Allowance for a new
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For some people living with rare diseases, changes in the ability to taste can affect much more than just food preferences. Loss of taste can impact nutrition, appetite, daily routines, and overall quality of life. Raising awareness of symptoms like these helps highlight the many ways rare diseases can affect the body and reminds us that even less visible symptoms can have a meaningful impact on everyday life.
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From our friends Cancer CommonsHere’s the latest from our Curious Dr. George! Read "The Strong Helping Hand of Cancer Commons" here: bit.ly/CCCDGBakker
Cancer is frequently accompanied by psychological distress and mental health challenges that are often neglected. Our Curious Dr. George asks Unsha Bakker, MSN, RN, OCN, CBCN, Cancer Commons Oncology Nurse Navigator for the Brain Cancer Support & Solutions Alliance (BCSSA), for her perspective on these issues—and how Cancer Commons can help.
BCSSA is a collaboration between Head For The Cure, the Musella Foundation For Brain Tumor Research & Information, Inc., and Cancer Commons. Our Nurse Navigator program is made possible through generous grant support provided by Head for the Cure and the Musella Foundation.
#CancerCommons #BCSSA #BrainCancerSupport #CancerNavigation #OncologyNurseNavigator #PatientSupport #CancerCare #MentalHealthInCancer #AdvancedCancer #PatientNavigation #BTSM
