Rare Diseases News Monitoring

Rare diseases in Malaysia: The hidden crisis few are talking about

One million Malaysians are affected by rare diseases, but the exact number remains unclear due to the lack of a national patient registry. Malaysia faces significant challenges in treating rare diseases, including limited resources, high treatment costs, …

House OKs boost for pediatric rare disease research

The House has cleared and sent to the Senate for consideration a bill that would increase research into pediatric rare diseases. The measure (HB 907), sponsored by Republican Rep. Adam Anderson, is dubbed the Sunshine Genetics Act. It would establish the …

2025 Rally for Rare: Bringing the Rare Disease Community Together

In a recent video interview with Applied Clinical Trials, Derek Ansel, vice president, therapeutic strategy lead, rare disease, Worldwide Clinical Trials, discussed the unique challenges in rare disease R&D, including competing priorities among …

Glycomine raises $115m to advance rare disease therapy into Phase IIb

Glycomine has raised $115m in Series C financing to advance its lead investigational therapy, GLM101, into a Phase IIb clinical trial for an ultra-rare disorder. GLM101 is being developed as a treatment for phosphomannomutase-2 congenital disorder of …

Monroe artist is advocate for rare diseases awareness

MONROE — When Giusiana Prosser paints, she doesn’t start with a sketch or a plan. She doesn’t even rinse her brush. Instead, she mixes emotion straight onto the canvas—using only black, white, and blue—and lets grief, resilience, and rage guide the strokes …

Transforming Rare Disease Drug Development For A Faster Cure

The swift journey of COVID-19 vaccines from gene discovery to mass deployment serves as a compelling precedent. Can this accelerated model be adapted to speed up the delivery of curative therapies for rare diseases? CDMOs are pivotal in this pursuit. To …

Peninsula biotech's megaround feeds clinical trial in devastating rare disease

The funding includes a number international venture capital firms and venture units of drug makers Novo Nordisk A/S, Sanofi SA and Asaha Kasei. Leaders of a small Bay Area company have long believed their way of replacing a piece of a sugar chain on …

Alzheimer’s and a rare disease: Everything we know about Gene Hackman and wife’s deaths

Your support helps us to tell the story From reproductive rights to climate change to Big Tech, The Independent is on the ground when the story is developing. Whether it's investigating the financials of Elon Musk's pro-Trump PAC or producing our …

Visalia man battles rare disease, family seeks funds for treatment

VISALIA, Calif. — Ernesto Olvera, a Visalia resident, has been fighting a rare autoimmune disease known as chronic inflammatory demyelinating polyneuropathy (CIDP) since December. The condition has left him paralyzed from the waist down and reliant on a …

Man's amazing tribute to best friend with rare disease

Paul Whitehead, 54 from Weymouth, is fundraising for Motor Neurone Disease Association by cycling from Lands End to John O’Groats, spanning three countries on foot and bike to raise awareness for people suffering with the life shattering disease. (Image: …

Peninsula biotech's megaround feeds clinical trial in devastating rare disease

Startup Glycomine raises $115M to push rare disease drug deeper into testing

Glycomine, a biotechnology startup working on a treatment for a rare genetic disorder, has raised a $115 million Series C round to push its lead program deeper into clinical testing. The funding announced Wednesday for the San Carlos, California-based …

Glycomine Gets $115M Series C for Rare Disease, Aims for Phase II Launch Mid-Year

California-based biopharma Glycomine announced a $115 million Series C raise Wednesday to push its lead rare disease molecule into Phase IIb trials. “We’re working on glycosylation disorders, putting sugar chains on proteins. There are hundreds of those,” …

Sarcoidosis awareness: UK HealthCare offers only clinic in Ky. for rare disease

LEXINGTON, Ky. (April 16, 2025) — Like many folks, Barry Pendleton had never heard of sarcoidosis.  So, he was shocked when he was diagnosed with the chronic, inflammatory disease that can negatively impact multiple systems throughout the body.   “Three, …

ChatGPT help woman diagnose 4-year-old son's rare disease after 17 doctors failed

New Delhi: In a remarkable story that demonstrates the expanding role of artificial intelligence in healthcare, an American woman turned to ChatGPT after 17 doctors failed to diagnose her four-year-old son's strange condition. The AI chatbot not only …

Rare Diseases: Opportunity for SC to Enable Access to Medicines

Two recent interim orders of the Supreme Court have pushed the lives of people living with rare diseases to the edge. These two orders were passed in two separate special leave petitions (‘SLP’) filed by the Union  challenging the High Court orders of  …

Researchers look at people with rare disease, find brain region involved in generosity

New Delhi: Studying a group of people with an affected ability to process emotions, researchers found a brain region that regulates generous behaviour -- the 'basolateral amygdala'. Driven by a concern for society, prosocial or altruistic behaviour …

Rare Disease Federal Funding Cuts | ‘This is What it Feels Like’ Season 2 | WWI Musical ‘The Hello Girls’

   Federal funding impacts to research for rare diseases. Also, the podcast “This is What it Feels Like” launches season two. Finally, the Women’s Theatre Collective’s new musical “The Hello Girls.” Rare Disease Federal Funding Cuts Since taking office, …

How one nonprofit is helping kids with rare diseases

There are over 7,000 rare diseases, and 25 to 30 million people in the U.S. are fighting one of these diseases every day. Many of those affected are children who need extensive medical care, and many of them are treated at home by their parents and family. …

Autism Acceptance Month Shines at Jay Nolan's Vibrant 'Picnic in the Park' Celebration

Families with children with disabilities enjoyed a day of fun, community, and support thanks to local sponsors and Jay Nolan Community Services LOS ANGELES, CA, UNITED STATES, April 16, 2025 /⁨EINPresswire.com⁩/ -- On Sunday, April 13, 2025, Jay …

Clean Remedies Lowers Tincture Prices to Make Wellness More Accessible

Clean Remedies, a wellness company known for its THC and CBD-infused products, has announced a recent price reduction across its line of oils and tinctures. AVON, OH, UNITED STATES, April 16, 2025 /⁨EINPresswire.com⁩/ -- Clean Remedies, a …

Hashimoto’s Disease and Other Thyroid Disorders: Treatment Program at Patients Medical

Hashimoto’s Disease: Comprehensive new program combines advanced thyroid testing, holistic care, and personalized hormone therapy for long-term results. NEW YORK, NY, UNITED STATES, April 16, 2025 /⁨EINPresswire.com⁩/ -- Patients Medical Unveils …

Post-TBI Symptom Management: Addressing Headaches, Mood Changes, and Mental Fatigue

METAIRIE, LA, UNITED STATES, April 16, 2025 /⁨EINPresswire.com⁩/ -- Traumatic Brain Injury (TBI) often marks the beginning of a prolonged recovery process that extends far beyond the initial trauma. Long after hospital discharge or acute care, many …

Naples Soccer Academy Launches Summer of Strength Program for Female Youth Athletes

In total, the program includes 29 gym-based training sessions and 10 field-based technical sessions, for a total of 39 high-impact sessions NAPLES, FL, UNITED STATES, April 16, 2025 /⁨EINPresswire.com⁩/ -- Naples Soccer Academy (NSA), a club- …

Exploring the Link Between Attention Deficit Disorder and Sensory Processing Sensitivity

GULFPORT, LA, UNITED STATES, April 16, 2025 /⁨EINPresswire.com⁩/ -- A growing body of research continues to explore the connection between Attention Deficit Disorder (ADD) and Sensory Processing Sensitivity (SPS), offering new insight into how …

Understanding Dental Insurance Coverage: Preventative, Basic, and Major Services Explained

HOUMA, LA, UNITED STATES, April 16, 2025 /⁨EINPresswire.com⁩/ -- Dental insurance continues to play an essential role in making oral healthcare more accessible and affordable across a broad population. From routine checkups to complex procedures, …

York Funding’s Peter Aytug and Rhea Stathatos Fund Development of 40,000 sq. ft. Factory in Bolivar, TN Industrial Park

New manufacturing facility contributing to job creation, technology innovation, and industry growth in Tennessee, is being financed by York Funding. NEW YORK, NY, UNITED STATES, April 16, 2025 /⁨EINPresswire.com⁩/ -- There is excitement and …

Growth Potential Consulting Honors Community Commitment Through Local Sponsorships

Jennifer L. Smith reaffirms her commitment to community impact through sponsorships supporting autism acceptance and student achievement. PRINCETON, NJ, UNITED STATES, April 16, 2025 /⁨EINPresswire.com⁩/ -- In celebration of its long-standing …

Empowering Local Chemists: A Call to Strengthen the Backbone of Community Healthcare

There are several individual medical stores that remain unorganized and lack proper training, support, service quality, & strategies to attract new customers. AHMEDABAD, GUJARAT, INDIA, April 16, 2025 /⁨EINPresswire.com⁩/ -- There are several …

Akelos Receives Australian Patent Allowance on Lead Compound, AKE-1018

Akelos -1018 for Chronic Neuropathic Pain NEW YORK, NY, UNITED STATES, April 16, 2025 /⁨EINPresswire.com⁩/ -- Akelos Receives Australian Patent Allowance on Lead Compound, AKE-1018 Patent Covers Inventions Licensed from Weill Cornell Medicine …

Beckerman Women’s Health Introduces Alma TED™ for Needle-Free Hair Restoration

Beckerman Women’s Health offers Alma TED™, a needle-free, pain-free treatment to restore hair growth and improve scalp health. ROCKVILLE, MD, UNITED STATES, April 16, 2025 /⁨EINPresswire.com⁩/ -- Hair loss is a widespread concern affecting …

New CDC Report Shows Higher Autism Rates and Highlights Complicated Challenges of Accurate Diagnosis

Expert Available for Interview WALNUT CREEK, CA, UNITED STATES, April 16, 2025 /⁨EINPresswire.com⁩/ -- The Centers for Disease Control and Prevention (CDC) yesterday released the 2025 Community Report on Autism. It found that 1 in 31 children in …

RAADfest 2025: Coalition for Radical Life Extension and Longevity Escape Velocity Foundation Join Forces

Join top longevity experts at RAADfest 2025 for breakthrough research and therapies, hosted with LEV Foundation and the Coalition for Radical Life Extension. LAS VEGAS, NV, UNITED STATES, April 16, 2025 /⁨EINPresswire.com⁩/ -- The LEV Foundation …

Bæredygtige og moderne træhuse: 3Huse ændrer boligspillet i 2025

3Huse sætter nye standarder i 2025 med moderne, bæredygtige træhuse bygget til helårsbrug – energieffektive, stilrene og klar til fremtiden. HELSINGøR, NORDSJæLLAND, DENMARK, April 17, 2025 /⁨EINPresswire.com⁩/ -- I 2025 er træhuse ikke længere …

Exokinetics Launches Project ELEVATE Mobility Foundation -- Empowering Those in Need of Mobility Solutions

PHILADELPHIA, PA, UNITED STATES, April 16, 2025 /⁨EINPresswire.com⁩/ -- Exokinetics, a leading mobility technology company and manufacturer of the groundbreaking Zeen mobility device, has officially launched its affiliate charitable foundation, …

Comfort Keepers Partners with Healthcare Providers to Enhance In-Home Care and Improve Client Outcomes

SHREWSBURY, NJ, UNITED STATES, April 16, 2025 /⁨EINPresswire.com⁩/ -- Comfort Keepers of Central New Jersey, a leading provider of in-home care services, is proud to announce its strategic partnerships with local healthcare providers to improve the …

Comfort Keepers Collaborates with Social Workers to Provide Specialized In-Home Care for Veterans

PITMAN, NJ, UNITED STATES, April 16, 2025 /⁨EINPresswire.com⁩/ -- Comfort Keepers in Southern New Jersey, a trusted provider of in-home care services, is pleased to announce an important collaboration with local social workers to enhance care for …

Society for Worldwide Medical Exchange Launches Initiative to Provide Clean Water to Schools Along the Marañón River

MIAMI, FL, UNITED STATES, April 16, 2025 /⁨EINPresswire.com⁩/ -- The Society for Worldwide Medical Exchange is pleased to announce a new initiative aimed at improving the health conditions of children living in communities along the Marañón River. …

Darwin Fitness offers Specialized Senior Training Program to Fight Osteoporosis & Improve Bone Density in Orlando FL

Darwin Fitness, Greater Orlando’s #1 private gym specialist in geriatric fitness, coaches older adults with specific bone health goals. ORLANDO, FL, UNITED STATES, April 16, 2025 /⁨EINPresswire.com⁩/ -- For seniors in the Orlando FL area seeking a …