Rare Disease
Maddie and the Drug that Turned Her Life Around
WebMD published a story about a young woman, Maddie, who had spent nine years in wheelchair confinement resulting from a rare metabolic disease related to
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When Breakthrough Drugs Need a New Home: Inside the Arvinas-Rigel Partnership
Claudine’s Journey with Parkinson’s Disease
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Cystinosis
Game-Changing Eye Drop Therapy Now Available to Canadian Cystinosis Patients
Health Canada has officially approved Cysklar® (cysteamine ophthalmic solution) 0.44%, marking a significant breakthrough for Canadians living with cystinosis. According to BusinessWire.com, the approval enables
Cervical Dystonia
Health Benefits of Putting Feelings to Words
Putting feelings into words does more than help you reflect. Brain imaging research shows it can shift activity inside emotional circuits. If you are someone
Rare Disease
“I Am More Than the Person in the Bed” – Kristian’s Message to the World
Most people see the bed first. That’s what defines me to them. The bed in the living room, the fact that I’m always in it.
Chronic urticarias
Enanta Begins First-in-Human Trial of Oral KIT Inhibitor for Chronic Urticaria
As reported by Business Wire, Enanta Pharmaceuticals has initiated its first clinical study of EDP-978, a novel oral inhibitor of the KIT receptor tyrosine kinase
Spinal Muscular Atrophy
“Not Defined By His Disability:” What Bennett Has Taught His Family About Spinal Muscular Atrophy
Editor’s Note: Patient Worthy is honored to share this article from our friend Jessica Lynn. To see the article in its original format, please click
Cancer
Targeting Cancer’s Hidden Killer: New Therapy Takes Aim at Muscle-Wasting Syndrome
Cancer cachexia represents one of oncology’s most overlooked catastrophes. Patients waste away, lose appetite, and deteriorate despite receiving chemotherapy, yet few treatment options exist to
Mucolipidoses (ML)
Throwback Thursday: Updates on Patients from Patient Worthy’s Past
I had learned that the many twists and turns on our rare disease path were similar to the families we met Originally published on May 11, 2022,
Bladder cancer
Jimi’s Fight Against Bladder Cancer
I was born in Long Island, New York, and raised in Southern California, where I grew up surrounded by sunshine and the beauty of the
Diabetic macular edema
A Six-Month Solution: How Eclipse Life Sciences Is Tackling a Persistent Vision Problem
Diabetic macular edema represents a silent threat to working professionals across America. The condition, which causes fluid accumulation in the macula, the eye’s central focusing
Lymphoma
ModeX Advances Novel Tetraspecific Antibody Into First-in-Human Study for B‑Cell Lymphoma
As reported on PharmaBiz, ModeX Therapeutics, a clinical-stage biotechnology company within OPKO Health, has begun dosing patients in a first-in-human clinical trial evaluating MDX2003, a
Malaria
Blood Supply, Malaria, and the Contribution of Grifols Pharma
A New Shield for the Blood Supply: Grifols Earns FDA Approval for Malaria Test Grifols has reached a significant milestone in transfusion medicine with the
Skin Disease
Galderma’s Latest Peptide Innovation Addresses a Critical Gap in Cosmetic Dermatology
The aesthetic skincare market has long overlooked a fundamental principle: skin preparation matters as much as the procedure itself. According to PharmaBiz.com, Galderma’s Alastin brand
Major Depressive Disorder
Motif Neurotech Gains FDA Clearance to Launch First-in-Human Trial of Implantable BCI for Depression
As reported on Business Wire, Motif Neurotech has received clearance from the U.S. Food and Drug Administration (FDA) to initiate its first clinical study evaluating
How to Transform Your Skin with Simple Natural Care Habits
For patients with rare diseases and the caregivers in healthcare who support them, skin changes often become a daily friction point rather than a simple
Pancreatic Cancer
Breakthrough in Pancreatic Cancer Treatment: How Revolution Medicines’ RAS Inhibitor Is Reshaping Patient Outcomes
Pancreatic cancer remains one of medicine’s most formidable challenges, and recent clinical developments suggest a potential turning point. Revolution Medicines presented compelling evidence this week
IgA Nephropathy (IgAN)
Ultomiris Reduces Proteinuria in Phase III Trial for IgA Nephropathy
Early Phase III data suggest C5 complement inhibition may alter disease course in IgAN In a recent press release, AstraZeneca reported that Ultomiris (ravulizumab) has
Rare Disease
Regular Yoga Practice Improves Cardiometabolic Health
Yoga is often celebrated for its ability to help people find their “zen,” but new research shows it might be doing some heavy lifting for
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Amyloidosis FoundationEnergy Conservation & Pacing With Amyloidosis
Adopt the "spoon theory" approach: view your energy as a limited set of spoons you must spend wisely each day.
Prioritize & Postpone: Focus on essential tasks and delay less critical ones for days when you feel stronger.
Take Scheduled Breaks: Instead of pushing until you are exhausted, take 5–10 minute rest breaks every hour or after 20–30 minutes of activity.
Sit When Possible: Use a stool or chair for everyday chores like showering, cooking, or folding laundry to reduce exertion.
Timing: Schedule demanding activities for times when you naturally have more energy, such as right after breakfast.
Children's Craniofacial Association"Cheers to Cher's 80th Birthday"
Cher's performance in the movie, Mask, released in 1985, sparked her interest in children and families affected by craniofacial differences. A few years later in 1990, she formalized her involvement with CCA, participating in Craniofacial Awareness Week in Washington, D.C. and signing on to become our national spokesperson. Cher conceived the idea of a camp for the whole family. Cher’s Annual Family Retreat was born. Since 1991, Retreats have been held annually throughout the country.
Watch a Video of Cher talking about CCA:
youtu.be/MpGR4Sdu4j8?si=iowFRmG-j38ENBuW
Donate to CCA in her honor:
ccakids.org/donate
Make a bid in her honor:
www.charitybuzz.com/categories/my-watches/catalog_items/auction-sestercentennial-412-carats-jewel...
@SumairaFoundation #nmo #neuromyelitisoptica
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