Rare Diseases News Monitoring

Dyno Therapeutics Launches Two New AAV Capsids and AI Platform for Rare Disease Therapeutic Development at the 2026 ...

WATERTOWN, Mass.--(BUSINESS WIRE)--May 13, 2026-- Dyno Therapeutics, Inc., a genetic technologies company applying artificial intelligence (AI) to solve the grand challenge of in vivo gene delivery, today announced the launch of two new adeno-associated …

Abeona pivots to T cells; AstraZeneca's rare disease drug passes Phase 3

AP High Court Directs Tirumala Tirupati Devasthanams To Consider Rare Disease, Autoimmune Disorder Patients For 'Special ...

The Andhra Pradesh High Court has directed the competent authority of Tirumala Tirupati Devasthanams (TTD) to decide within four months a representation seeking expansion of 'Special Darshan' medical quota to devotees having debilitating illnesses …

RTW Foundation Awards $573,000 to Rare Disease Foundations Advancing Ultra-Rare Disease Research

RTW Foundation is proud to announce $573,000 in grant funding for eight rare disease foundations advancing critical rare disease research. NEW YORK CITY, NY, UNITED STATES, May 12, 2026 /⁨EINPresswire.com⁩/ -- RTW Foundation, the philanthropic arm …

Repair Biotechnologies Granted Rare Disease Evidence Principles (RDEP) Eligibility by the FDA for REP-0003 in Homozygous ...

SYRACUSE, NY / ACCESS Newswire / May 12, 2026 / Repair Biotechnologies, Inc., a biotechnology company developing first-in-class therapies capable of rapidly regressing atherosclerotic plaque, today announced that the U.S. Food and Drug …

3-year-old with rare disease helps raise $500K for inclusive playground

A 3-year-old with a rare condition has helped raise about $500,000 to build an inclusive playground at his school. Bedford Erickson, 3, has captured the hearts of hundreds of thousands of followers on social media who follow his journey living with a rare …

BGI Genomics joins HGP2 Rare Disease Alliance to close rare disease care gap in Asia-Pacific

May 12 2026 The HGP2 Rare Disease Alliance of the Asia-Pacific Region (HGP2 RaDiAnce–APAC) was officially launched in Kuala Lumpur on May 10.  YBhg. Datuk Dr. Nor Fariza Binti Ngah, Deputy Director-General of Health (Research and Technical Support) at the …

Moving Film “Dee & Dan” Documents a Day Living With Niemann-Pick Disease as an Intimate Portrait of Resilience

CHICAGO & NEW YORK & SAN DIEGO--(BUSINESS WIRE)--May 13, 2026-- Dee & Dan, a powerful new short film, debuts today as the first production in the In the Light film series from 24/364, a cultural brand driving awareness, connection, and …

Catalent and Elpida Therapeutics Enter Strategic Partnership for Late-Phase AAV Manufacturing

TAMPA, Fla. & CAMBRIDGE, Mass.--(BUSINESS WIRE)--May 13, 2026-- Elpida Therapeutics, a non-profit biotechnology company developing gene therapies for ultra-rare diseases, and Catalent, Inc., the leader in enabling the development and supply of better …

Roche, MET to open disease treatment hub

International biotech company Roche Pharmaceuticals and Medical Excellence Taiwan (MET) yesterday announced the launch of the Asia Pacific Hub for Rare Disease Innovation and Treatment, signing a letter of intent under the witness of the Ministry of Health …

WATCH: Longford siblings with rare disease who 'don't have time' issue HSE plea for drug

Two North Longford siblings battling a rare, progressive neurodegenerative disease who are campaigning for access to life-changing treatment for them and 200 patients, have urged the government and drug manufacturer Biogen, to come together to find a …

✰PREMIUM Co-creator of ‘How I Met Your Mother’ signs books and discusses parenting a son with a rare disease

Having a child with a disability also exposed the couple to a community of devoted parents and school staff. Elliot attended the Ideal School in Manhattan, which was founded by Michelle Smith, Audra Zuckerman-Rubin and Mitch Rubin, three parents of …

STAT+: Astellas treatment offers new hope to a devastated rare disease community

Want to stay on top of the science and politics driving biotech today? Sign up to get our biotech newsletter in your inbox. There’s fresh turmoil at the very top of the FDA: All weekend, questions have swirled over whether Commissioner Marty Makary will be …

'Wondrous' girl, 11, died after rare disease therapy left her body too weak to fight infection

Elsa Pope died at the infirmary on Christmas Day, 2024(Image: Leicester Mercury / Chris Gordon) A "wondrous" young girl died on Christmas Day from a fungal infection after treatment for a rare condition weakened her immune system, an inquest …

Alliance launched in Asia to enhance rare disease healthcare

Kuala Lumpur, May 11: The Human Genome Project II Rare Disease Alliance of the Asia-Pacific Region (HGP2 RaDiAnce-APAC) was launched here on Sunday to advance regional collaboration in rare disease care. The launch meeting brought together clinical experts …

Development Trends of Janus Kinase Inhibitors (JAKi) Over Three Decades: From Common to Rare Diseases

Introduction The Janus Kinase-Signal Transducer and Activator of Transcription (JAK/STAT) signaling pathway is a key regulator of cytokine communication. It is activated by multiple cytokine interactions with cell membrane receptors, leading to JAK …

Alliance launched in Malaysia to enhance regional collaboration in rare disease care

KUALA LUMPUR, May 11 (Xinhua) -- The Human Genome Project II Rare Disease Alliance of the Asia-Pacific Region (HGP2 RaDiAnce-APAC) was launched here on Sunday to advance regional collaboration in rare disease care. The launch meeting brought together …

India’s genetic mosaic: how understanding our genes can help improve our health

India is home to more than 1.4 billion people, thousands of communities, hundreds of languages, and five major language families. Many communities have historically practised endogamy and in some regions, consanguineous marriage. This is both a social and …

The Hantavirus Panic Machine: When Rare Diseases Become Media Theater

Periodically, the public faces a new microbial threat. The pattern is consistent: a tragic death or cluster of illnesses emerges, prompting newsrooms to employ dramatic language such as “deadly virus,” “mysterious outbreak,” and “health officials concerned …

Own Your Sleep™ Launches Travel Comfort Kits for Medical, Sensory, and Everyday Travelers

Michigan-founded company introduces medically relevant travel comfort kits and the world's first hotel certification program for adjustable beds. WATERFORD, MI, UNITED STATES, May 13, 2026 /⁨EINPresswire.com⁩/ -- Own Your Sleep™ Launches …

Assisting Hands Home Care Expands Awareness of Personalized In-Home Care Services for Families in Ashburn, Virginia

Assisting Hands Home Care provides home care in Ashburn, including companion care, personal care, dementia care, live-in care, and 24-hour support. ASHBURN, VA, UNITED STATES, May 13, 2026 /⁨EINPresswire.com⁩/ -- Assisting Hands Home Care, a …

MERIT Announces Strategic Collaboration with MTI to Provide Spirometry Endpoints

For Too Long, Spirometry Had One Way In. Not Anymore. MADISON, WI, UNITED STATES, May 13, 2026 /⁨EINPresswire.com⁩/ -- MERIT CRO, Inc. (MERIT) has partnered with Monitored Therapeutics, Inc. (MTI) to offer a new standard for spirometry endpoints—combining …

The Sheri and Les Biller Family Foundation Launches 'Support Is Care' Campaign

Transforming the Cancer Patient Experience SEATTLE, WA, UNITED STATES, May 13, 2026 /⁨EINPresswire.com⁩/ -- With nearly 40% of Americans expected to face a cancer diagnosis in their lifetime, the journey through treatment remains one of the most …

Jazzy Day Spa Introduces Glacē™ Treatment: A Gentle, Skin-Purifying Ritual for a Grounding, Glowing Complexion

SOUTH MIAMI, FL, UNITED STATES, May 13, 2026 /⁨EINPresswire.com⁩/ -- Jazzy Day Spa, a leading provider of advanced aesthetics and skincare solutions in Miami, FL, is proud to announce the launch of Glacē™ treatment, a gentle, multi-step …

PLEXIS Healthcare Systems Highlights Claims Automation to Help Payers Improve Accuracy and Reduce Operational Burden

Payers are increasing automation at intake, but many still struggle to achieve accurate, end-to-end claims processing without downstream rework MEDFORD, OR, UNITED STATES, May 13, 2026 /⁨EINPresswire.com⁩/ -- PLEXIS Healthcare Systems today …

The Ginger Network Launches New Website

Adding “More Ginger” Expertise for Food, Nutrition, Health & Wellness Partners ARLINGTON, VA, UNITED STATES, May 13, 2026 /⁨EINPresswire.com⁩/ -- The Ginger Network (TGN), a premier marketing communications consultancy dedicated exclusively to …

United Cerebral Palsy Conference Gathers Leaders, Advocates, and Innovators to Advance Disability Inclusion and Services

UCP’s Annual Conference explored innovative solutions, addressed challenges in disability services, and advanced inclusion for people with disabilities. VIENNA, VA, UNITED STATES, May 13, 2026 /⁨EINPresswire.com⁩/ -- United Cerebral Palsy (UCP …

Intracranial Stents Market: Size, Trends, and Strategic Outlook (2026–2033)

The intracranial stents market is growing due to rising cerebrovascular diseases, minimally invasive procedures, and advanced neurovascular technologies. LONDON, UNITED KINGDOM, May 13, 2026 /⁨EINPresswire.com⁩/ -- The global intracranial stents …

IgA Nephropathy Foundation Launches 'Are You IgAN Aware?' PSA Campaign

The Foundation marks its 22nd anniversary with “Strike Out IgAN,” a national awareness PSA, and recognition at the ballpark NJ, UNITED STATES, May 13, 2026 /⁨EINPresswire.com⁩/ -- IgA Nephropathy Foundation Celebrates 22 Years of Impact, Launches …

Presenting: Virginia’s Innovation Crossroads

New brand identity is ‘Built for What’s Next’ in greater Fredericksburg region FREDERICKSBURG, VA, UNITED STATES, May 13, 2026 /⁨EINPresswire.com⁩/ -- The Fredericksburg Regional Alliance (FRA), an organization that has proudly served as one of …

PCOS Renamed to PMOS—HRT University Had It Right All Along

A landmark Lancet consensus study has renamed PCOS to PMOS, validating a metabolic framework that HRT University has built into its accredited curriculum. CINCINNATI, OH, UNITED STATES, May 13, 2026 /⁨EINPresswire.com⁩/ -- When a landmark study …

Garland, Texas Family Dental Practice Marks Recognition Among Region's Top Providers

Texas dentist Dr. John Paul, named to D Magazine's 2026 Best Dentists list by peers, runs a three-location practice serving Medicaid and CHIP families. GARLAND, TX, UNITED STATES, May 13, 2026 /⁨EINPresswire.com⁩/ -- GARLAND, Texas. A family …

Net Health Names Wound Care Advantage a First-Ever Momentum Award Winner

Wound Care Advantage wins the first-ever Net Health Value of Innovation Award for Wound Care, recognized for Luvo, wound care software built for hospitals SIERRA MADRE, CA, UNITED STATES, May 13, 2026 /⁨EINPresswire.com⁩/ -- Wound Care Advantage ( …

Blood Brain Barrier Technology Market: Global Insights, Trends, and Forecasts

The blood brain barrier technology market focuses on innovative drug delivery systems for neurological and oncological therapies worldwide. LONDON, UNITED KINGDOM, May 13, 2026 /⁨EINPresswire.com⁩/ -- The global blood brain barrier (BBB) …

Lifelong Union Member Furhan Ahmad Earns Major Labor Endorsements in Assembly District 66 Race

UFOA, UFO and Marine Engineers Back First Responder and Lifelong Union Member for Lower Manhattan Assembly Seat NEW YORK, NY, UNITED STATES, May 13, 2026 /⁨EINPresswire.com⁩/ -- Furhan Ahmad, Democratic candidate for New York State Assembly …

Why the Best Scrub Caps for Women Are Driving a Shift in Healthcare - What Medical Professionals Are Demanding in 2026

Discover why the best scrub caps for women are driving demand for better fit, comfort, and performance during long shifts in modern healthcare settings. AUSTIN, TX, UNITED STATES, May 13, 2026 /⁨EINPresswire.com⁩/ -- A quiet but meaningful …

SCS Skincare Solutions Elite Medispa Named 2026 Allergan Top 500 Award Winner, Top 1% of Aesthetic Practices in the U.S.

SCS Skincare Solutions Elite Medispa is proud to be named a 2026 Allergan Top 500 Award Winner, in the top 1% of aesthetic providers in the US. UNIONTOWN, OH, UNITED STATES, May 13, 2026 /⁨EINPresswire.com⁩/ -- SCS Skincare Solutions Elite MediSpa is …

RoboCT Welcomes Peer-Reviewed Study on Exoskeleton Gait Rehabilitation After Stroke

Peer-reviewed study supports a more evidence-based approach to exoskeleton-assisted gait rehabilitation after stroke. HANGZHOU, ZHEJIANG, CHINA, May 13, 2026 /⁨EINPresswire.com⁩/ -- RoboCT Technology Development Co., Ltd. today welcomed the …

Artificial Intelligence (AI) Dermatology Imaging Market to Reach $2.9B by 2030, Growing at 21% CAGR Between 2026–2030

The Business Research Company's Artificial Intelligence (AI) Dermatology Imaging Market Report 2026 – Market Size, Trends, And Global Forecast 2026-2035 LONDON, GREATER LONDON, UNITED KINGDOM, May 13, 2026 /⁨EINPresswire.com⁩/ -- "The …