Gene therapy trial offers breakthrough for rare genetic disorder

Published Date: 26 Jul 2025

A study evaluating a pioneering lentivirus (LV)-mediated gene therapy trial for classical Fabry disease showed promising results over five years, indicating a potential breakthrough in treatment for the genetic disorder.

Read Full News

Latest News

US Provisional Mortality Rate Falls 4.6% in 2025, Flu Deaths Rise

The U.S. provisional death rate fell 4.6% in 2025, with heart disease, cancer and unintentional injuries remaining the leading causes of mortality, ⁠the Centers for Disease...

Bangladesh Warns Of Dengue Surge as Weather Aids Spread

Bangladesh could face a sharp increase in dengue infections over the next two months, health experts warned, as wet ⁠weather and inadequate mosquito control spur a wider...

Uganda Finds Isolated Marburg Virus Case, Africa CDC Says

Ugandan health authorities have confirmed an isolated case of Marburg virus disease, a highly infectious hemorrhagic ⁠fever, while conducting surveillance for an Ebola...

WHO Recommends Doxycycline PEP to Prevent Bacterial STIs

WHO’s first guidance on doxyPEP prioritizes high-risk groups while emphasizing antimicrobial resistance monitoring and responsible implementation.

Congo Bans Gatherings in Kinshasa and Three Provinces Over Ebola Outbreak

The Democratic Republic of Congo has banned public gatherings in four provinces, including the capital, Kinshasa, as ⁠the country battles a deadly Ebola...

Ebola Outbreak Could Cost Africa Up to $3.6 Billion, UN Says

(Edits headline to spell out 'billion')

Todays Updates

Enhancing Comprehensive Biomarker Testing and Biomarker-Directed Treatment Selection

Enhancing Comprehensive Biomarker Testing and Biomarker-Directed Treatment Selection

Advancing Care in ES-SCLC: Personalized Strategies, New Therapies, and Real-World Insights

Advancing Care in ES-SCLC: Personalized Strategies, New Therapies, and Real-World Insights

Adjuvant Combination for Kidney Cancer Gets FDA's Blessing

Belzutifan and pembrolizumab together reduced the risk of disease recurrence or death

FDA Approves Prostate Cancer Combo That Warranted Advisory Meeting

Capivasertib plus abiraterone combination lands indication for PTEN-deficient tumors

Talquetamab Regimens Boost Survival in Multiple Myeloma

"Combinations represent additional options for early-line treatment," researcher says

In High-Risk B-Cell ALL, Swapping Out Chemo for Blinatumomab Improves Outcomes

The bispecific T-cell engager also reduced toxicity in newly diagnosed kids

Timely 'Nudges' Can Help Encourage More End-of-Life Conversations in Cancer

A combination of clinician and patient prompts worked best, but talks remained infrequent

Todays Updates

Enhancing Comprehensive Biomarker Testing and Biomarker-Directed Treatment Selection

Enhancing Comprehensive Biomarker Testing and Biomarker-Directed Treatment Selection

Advancing Care in ES-SCLC: Personalized Strategies, New Therapies, and Real-World Insights

Advancing Care in ES-SCLC: Personalized Strategies, New Therapies, and Real-World Insights

Adjuvant Combination for Kidney Cancer Gets FDA's Blessing

Belzutifan and pembrolizumab together reduced the risk of disease recurrence or death

FDA Approves Prostate Cancer Combo That Warranted Advisory Meeting

Capivasertib plus abiraterone combination lands indication for PTEN-deficient tumors

Talquetamab Regimens Boost Survival in Multiple Myeloma

"Combinations represent additional options for early-line treatment," researcher says

In High-Risk B-Cell ALL, Swapping Out Chemo for Blinatumomab Improves Outcomes

The bispecific T-cell engager also reduced toxicity in newly diagnosed kids

Timely 'Nudges' Can Help Encourage More End-of-Life Conversations in Cancer

A combination of clinician and patient prompts worked best, but talks remained infrequent

Latest News

The U.S. provisional death rate fell 4.6% in 2025, with heart disease, cancer and unintentional injuries remaining the leading causes of mortality, ⁠the Centers for Disease...

Bangladesh could face a sharp increase in dengue infections over the next two months, health experts warned, as wet ⁠weather and inadequate mosquito control spur a wider...

Ugandan health authorities have confirmed an isolated case of Marburg virus disease, a highly infectious hemorrhagic ⁠fever, while conducting surveillance for an Ebola...

WHO’s first guidance on doxyPEP prioritizes high-risk groups while emphasizing antimicrobial resistance monitoring and responsible implementation.

The Democratic Republic of Congo has banned public gatherings in four provinces, including the capital, Kinshasa, as ⁠the country battles a deadly Ebola...

(Edits headline to spell out 'billion')

News All Time

WHO Says Congo's Ebola Outbreak Still Fast-moving and Health Workers Under Threat

The Ebola outbreak in Democratic Republic of Congo is still outpacing response efforts, and frontline health workers there ⁠face very risky conditions, World Health...

UNICEF, Gavi Call to Speed Bundibugyo Ebola Vaccine Access

UNICEF and global vaccine group Gavi said on Wednesday they are seeking information from developers and manufacturers ⁠on plans for a vaccine against the Bundibugyo strain...

Cytokine Scaffold Builds Long-Lived CAR T Cells Against HIV

A new approach aims to use CAR T cells to last longer in the fight against HIV.

Ebola Infects More Than 1,000 in Congo, Spreads to Kill Toddler in New Camp

Ebola has now infected more than 1,000 people in the Democratic Republic of Congo in ⁠an outbreak that has spread to a third displacement camp and killed an 18-month-old...

Pfizer's Experimental Drug Misses Main Goal Of Lung Cancer Trial

Pfizer said on Monday that one of the key experimental drugs it picked up in its $43 billion 2023 acquisition of ⁠Seagen failed to improve survival when compared to...

UNAIDS Chief Urges US to Reconsider South Africa Funding Cut

The head of UNAIDS said on Monday she was saddened by U.S. plans to withdraw HIV/AIDS funding for South Africa and urged Washington to reconsider, warning the move could...

UN Agencies Launch $1 Million Project to Contain Screwworm Outbreak

The International Atomic Energy Agency and the U.N. food agency have launched a $1 ⁠million research project to help contain the screwworm outbreak in the Americas,...

Inghams Locks Down Farms After Australia Reports Second H5N1 Case

Inghams Group on Monday implemented a complete lockdown across all of its Western ⁠Australian farms and processing operations after Australia reported a second case of the...

USDA Reports Three New Cases of Screwworm, Bringing Total to 15

The number of U.S. cases of New World screwworm has risen to 15 after three ⁠more animals tested positive in Texas, the Department of Agriculture said in a post on social...

Simple Molecular Test May Detect Pneumocystis Pneumonia

A simple PCR of mouth rinse may serve as a useful non-invasive test in diagnosing Pneumocystis pneumonia compared with bronchoalveolar lavage in immunocompromised patients without HIV.

Australia, Last Continent Without H5 Bird Flu, Detects First Suspected Case

Australia has detected its first suspected mainland case of H5N1 bird flu in a remote part of the country's southwest, ⁠authorities said on Friday.

Congo Says Number Of Confirmed Ebola Cases Rises to 896

The number of confirmed Ebola ⁠cases in the Democratic Republic of Congo ⁠has increased to 896, including 232 ⁠deaths, government ‌data showed late on...

US FDA Advisers Vote in Favor of Moderna's Flu Vaccine

The U.S. Food ⁠and Drug Administration's ⁠advisers on Thursday backed ⁠approval ‌of Moderna's flu vaccine in adults aged 50 and ‌older, saying its benefits outweigh ‌the...

Oldest-known Plague Outbreak Came 5,500 Years Ago in Siberia

About 5,500 years ago, bands of hunter-gatherers inhabited the Lake Baikal region in Siberia, sustained by rich resources including prey such as elk, deer, moose, fish,...

Ebola Bundibugyo Vaccine Candidates Could Enter Phase 1 Trials as Early as July

(Refiles to fix typo in ⁠country name)

G7 Leaders Call for Strong, Coordinated Response to Ebola Outbreak

G7 leaders called ⁠on Tuesday for a strong and ⁠coordinated response to the Ebola ⁠outbreak in ‌Congo, urging other nations to dedicate resources in a bid to ensure ‌the...

Recommended News For You

FDA Leaders Explain Their Choice Regarding Cancer Risk Warning in CAR-T Investigation.

Alkem introduces cetuximab under the trade name Cetuxa in India.

Hematocrit Stabilized in Polycythemia Vera Under Novel Hepcidin Mimetic Treatment.

PNH With Extravascular Hemolysis Approves Factor D Inhibitor.

Genetics and Genetic Testing to Inform Myelofibrosis Clinical Management.

Indoor hydroponic gardening can improve mental health and quality of life for cancer patients

Skipping Lymph Node Biopsy Gains Ground in Breast Cancer

Recommended Articles For You

CAR T + Ibrutinib in R/R Mantle Cell Lymphoma: Phase 2 TARMAC Study Insights 

Unlocking the Potential of Hemin: A Novel Approach to Health and Wellbeing 

Value, Access, and Affordability in Next-Gen Oncology: Navigating Costs, Coverage, and Clinical Impact 

Breakthroughs in Oncology: Advances in Immunotherapy, Radiation & Precision Diagnostics 

Whats more on Hidoc Dr.

Medical Updates
KOL Videos
Surveys
Events

Daily news to keep you up to date

Gain valuable insights with our in-depth KOL Video discussions

Participate and win cash vouchers

Get updates on the latest events happening around the world
Featured News
Featured Articles
Featured Events
Featured KOL Videos

1.

AI model combines clinical and magnetic resonance data to improve prediction of breast cancer recurrence

2.

'It's rare and it's scary.' Dark spot on your fingernail could mean cancer

3.

24 months after radical prostateectomy, no PSA recurrence was observed when using apalutamide plus ADT.

4.

Modified personalized cancer vaccine generates powerful immune response

5.

Spouses of Cancer Patients May Be at Increased Risk of Suicide

1.

Insights into Doxorubicin and Liposomal Doxorubicin in Extra-Abdominal Desmoid-Type Fibromatosis

2.

Mastering Surgical Oncology: Education, Certification, Trials, and Therapy Insights

3.

The Millionaire's Battle: Does Mark Cuban Have Cancer?

4.

Don't Ignore Your Vision Loss: What You Need to Know About Amaurosis Fugax 

5.

Evidence-Based Insights in Oncology for Specialists

1.

Asian Symposium on Advancement in Hematology and Oncology

2.

Asian Symposium on Advancement in Hematology and Oncology

3.

Asian Symposium on Advancement in Hematology and Oncology

4.

International Cancer Conference

5.

Asian Symposium on Advancement in Hematology and Oncology

1.

Navigating the Complexities of Ph Negative ALL - Part IX

2.

Treatment Sequencing Strategies in ALK + NSCLC Patients with CNS Diseases

3.

Navigating the Complexities of Ph Negative ALL - Part XIV

4.

Experts' Opinion on the Goal of Treatment of Patients with Relapsed Adult B-cell ALL

5.

An In-Depth Look At The Signs And Symptoms Of Lymphoma

Fastest Growing Medical Platform for Doctors

Address :
Hidoc Dr. Inc. | Delaware C Corp | 1309 Coffeen Ave. Suite 1200, Sheridan WY, 82801

Phone :
+1-415-463-3094

Email :
anishagadia@hidoc.co

Quick Links

Drug DatabaseDrug InteractionDiseases Database
KOL VideosKnowledge HubQuizzes
Medical UpdatesEventsAbout Us

© Copyright 2026 Hidoc Dr. Inc.

Terms & Conditions - LLP | Inc. | Privacy Policy - LLP | Inc. | Account Deactivation
Home
Profile
Login
bot