Leigh syndrome
New Hope for Leigh Syndrome
As reported on Inside Precision Medicine, scientists in Germany have made an exciting discovery that could help children with a very rare and serious illness
Can We Fix This Broken Heart? – Part 1
Draft Guidance from FDA Introduces Important Changes to Drug Development for Rare Diseases
How I Manage My Pain – Pain Series Part 3 of 3
LISTEN TO OUR PODCAST
THE LATEST FROM PATIENT WORTHY
Lung Cancer
Regulatory Fast-Track Transforms Lung Cancer Treatment Timeline; Gene Therapy Withdrawal Signals Market Challenges
The biotechnology sector witnessed a striking demonstration of expedited drug development this week, with Boehringer Ingelheim receiving FDA approval for an innovative lung cancer therapy
Rheumatoid Arthritis
Elevara Advances Oral CDK4/6 Inhibitor into Mid-stage Rheumatoid Arthritis Trial
As reported by Pharma Times, Elevara Medicines has initiated a phase 2b clinical study evaluating ELV001, an investigational oral CDK4/6 inhibitor, in people with rheumatoid
Triple-negative breast cancer
Dark Sweet Cherries Against Breast Cancer
Pictured above: Giuliana Noratto, Ph.D., (left) and Lauren Stranahan, Ph.D., led research showing that compounds in dark sweet cherries may help slow the growth and
Breast Cancer
Japan Advances Targeted Breast Cancer Treatment: Daiichi Sankyo Seeks Approval for Innovative Therapy After Surgery
Daiichi Sankyo has taken a significant step toward expanding treatment options in Japan by submitting regulatory approval for Enhertu, an advanced cancer-fighting drug designed to
Asthma
FDA Clears First Independent Generic of GSK’s Flovent Asthma Inhaler
As reported by FiercePharma, the U.S. Food and Drug Administration has approved the first fully independent generic version of GlaxoSmithKline’s widely used asthma inhaler, Flovent,
Trimethylaminuria
My Journey, My Life, My Story, My Book – Carry’s Experiences with “Fish Odor Syndrome”
You can not see, hear, or touch my rare condition. I STINK! (LITERALLY) I wasn’t sure if I was going to tell my story. But
HIV/AIDS
Scientists Redesign HIV-Fighting Antibodies to Strike Faster: A New Engineering Breakthrough
Researchers have created a fundamentally new class of HIV-fighting molecules by combining two separate antiviral mechanisms into a single engineered protein. Early laboratory results, reported
Psoriatic Arthritis
Bimzelx Outperforms Skyrizi in Psoriatic Arthritis Trial, Though Commercial Impact Remains Unclear
As reported on BioPharmaDive, UCB has reported positive results from a head-to-head clinical trial showing that its anti-inflammatory biologic Bimzelx was more effective than AbbVie’s
Colorectal cancer
Skipping A Colonoscopy Almost Cost Me Everything
Editor’s Note: Patient Worthy is proud and honored to share Jennifer’s story with cystic fibrosis and colon cancer, originally published on the Cystic Fibrosis Research
Seizures
Momentum Builds Across Biotech Pipeline: Seizure Breakthrough, Cell Therapy Revival, and Obesity Medicine Gains
The biotechnology sector experienced significant positive developments this week, with Xenon Pharmaceuticals’ seizure medication achieving a decisive late-stage victory while regulatory pathways accelerated for several
Breast Cancer
GLP-1 Receptor Agonists and Breast Cancer: Promising Signals, Pressing Questions
As reported on Healio, glucagon-like peptide-1 (GLP-1) receptor agonists, best known for treating diabetes and obesity, are emerging as unexpected players in breast cancer care.
Triple-negative breast cancer
Study: Engineered Immune Cells and Targeted Treatments May Slow the Spread of Triple-Negative Breast Cancer
CAR T- cells are immune cells that are engineered in a laboratory to attack cancer. The therapy has worked as planned for certain blood cancers
BRCA-mutated Breast Cancer
Breakthrough DNA Repair Inhibitor Advances Rapidly Toward Clinic: Artios’ ART6043 Earns FDA Fast Track Status for Resistant Breast Cancer
Artios Pharma has achieved a significant milestone in cancer treatment development with the U.S. Food and Drug Administration’s granting of Fast Track designation to ART6043,
Multiple Myeloma
Bristol Myers Squibb Reports Positive Phase 3 Data for Mezigdomide-Based Oral Regimen in Relapsed or Refractory Multiple Myeloma
As reported on drugs.com, Bristol Myers Squibb (BMS) has announced encouraging interim results from the Phase 3 portion of the SUCCESSOR-2 clinical trial evaluating oral
Hunter Syndrome
FDA Explains Rejection of Regenxbio’s Hunter Syndrome Gene Therapy
As reported on FierceBioTech, the U.S. Food and Drug Administration has released its complete response letter (CRL) detailing why it declined to approve Regenxbio’s experimental
Breast Cancer
Roche’s Phase III persevERA Trial of Giredestrant Misses Primary Endpoint in Advanced ER-Positive Breast Cancer
As covered on PharmaBiz, Roche has reported topline results from the phase III persevERA Breast Cancer study, which evaluated the oral selective oestrogen receptor degrader
Rare Disease
BASELINE
Editor’s Note: Patient Worthy is proud to share this article from our friends at the Courageous Parents Network. A parent often can sense when there
FEATURED
UPCOMING EVENTS
SIGN UP FOR OUR NEWSLETTER
Throughout the week, Patient Worthy is highlighting HIV with educational content aimed at raising awareness and fostering greater understanding in our community.
We’ll be sharing information on our socials and patientworthy.com to educate, empower, and support those affected.
If you'd like to share your story with HIV, click here: bit.ly/4dV7gru
#HumanImmunodeficiencyVirus #HIV #HIVAwareness #ShareYourStory #PatientWorthy
April 5th is Barth Syndrome Awareness Day - a time to shine a light on a rare genetic condition that affects the heart, muscles, and immune system. Barth Syndrome may be a little known, but for those living with it, the challenges are very real. Raising Awareness helps drive research, early diagnosis, and support for families who need it most.
Every voice matters. By learning, sharing, and advocating, we can help create a future where rare diseases like this are better understood and treated. #barthsyndromeawareness #geneticdisorders #PatientWorthy
Head and neck cancers affect vital parts of how we speak, breathe, and connect with the world- yet many people don't recognize the early warning signs. Persistent sore throat, difficulty swallowing, voice change, or unusual lumps should never be ignored. Early detection can save lives.
Awareness isn't just knowledge- it's action. Protect your health by avoiding tobacco, limiting alcohol, considering HPV vaccination, and seeking medical advice when something feels off. Your voice matters- take care of it.
#headandneckcancer #preventcancer #PatientWorthy
