Rare disease news, mapped to the diseases behind the headlines. AI-classified across pipeline, policy, funding, science, and community.
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Friday, February 6
FDA Launches START Program to Accelerate Rare Disease Therapies
FDA Targets Non-Approved GLP-1 Drugs, Impacting Rare Disease Access
Biotech IPOs Surge as Agomab and Eikon Raise Over $580M This Week
First Personalized Gene Therapy Successfully Administered to Infant with Rare Disease
Rare Disease Day Amplifies Advocacy Impact and Awareness Initiatives
We pull from 25+ sources — FDA, NIH, clinical registries, top trade publications, and SEC filings.
AI reads every article, assigns an executive category, scores importance, and writes a 2-sentence summary.
Each story is auto-tagged to specific rare diseases in our 15,964+ disease taxonomy — so you see what matters to you.
Showing 1–20 of 2528 stories
The Mental Health and MAMA Act of 2026 has been referred to multiple committees for consideration. This legislation aims to address mental health issues, although specific provisions are yet to be detailed.
The Senate has introduced bill S 1055 to amend the Indian Health Care Improvement Act, focusing on modifying notification requirements for emergency contract health services. Hearings have been held by the Committee on Indian Affairs.
The Senate has introduced bill S 3712, which mandates a study on the use of home cardiorespiratory monitors for infants. The bill has been read twice and is currently under review by the Committee on Health, Education, Labor, and Pensions.
SJRES 103 aims to disapprove a rule from the Department of Veterans Affairs concerning reproductive health services. The resolution has been read twice and is currently under review by the Committee on Veterans' Affairs.
The bill S 2771 aims to amend the Indian Self-Determination Act and the Indian Health Care Improvement Act to provide advance appropriations authority for specific accounts within the Bureau of Indian Affairs, Bureau of Indian Education, and Indian Health Service. It has been read twice and referred to the Committee on Indian Affairs.
The Senate has introduced bill S 699 to amend the Indian Health Care Improvement Act, focusing on liability for payment of charges related to purchased or referred care services. Hearings have been held by the Committee on Indian Affairs.
A case report highlights a rare instance of epithelioid and spindle cell rhabdomyosarcoma of the rib associated with a FUS-TFCP2 fusion. This adds to the understanding of genetic factors in this rare cancer subtype.
A case report details a massive non-functioning adrenocortical carcinoma that presented as an acute abdomen without rupture. This case highlights the complexities of diagnosing rare tumors and the need for awareness in clinical settings.
The Department of Health and Human Services has re-established a matching program between CMS and the Social Security Administration to determine eligibility for Qualified Health Plans. This program aims to streamline the enrollment process and issue certificates of exemption.
Research misconduct findings against Dr. Daniel Andrade highlight issues in grant applications for U.S. Public Health Service funds. This incident may impact future funding and research integrity policies.
The Senate has ordered S 2546 to be reported favorably, extending the legislative authority of the National Emergency Medical Services Memorial Foundation for a commemorative work in Washington, D.C. This bill reflects ongoing support for emergency medical services.
A recent study published in PubMed provides insights into the clinical characteristics and natural history of arginase 1 deficiency, based on data from international clinical trials. This research enhances understanding of the disease's progression and patient outcomes.
A recent case report and systematic review highlight the rare occurrence of colonic metastasis from breast carcinoma. This study provides insights into the clinical implications and management of this uncommon metastatic pattern.
A case report highlights the challenges of performing coronary artery bypass grafting in patients with severe hyperthyroidism. This study contributes to understanding the surgical risks associated with this endocrine disorder.
The FDA plans to restrict non-FDA-approved GLP-1 drugs, targeting compounded medications that are being mass-marketed. This action could significantly impact the availability of these drugs in the market.
HR 3413, known as the Physician and Patient Safety Act, has been referred to the House Committee on Energy and Commerce. This bill aims to address safety concerns for both physicians and patients.
A review highlights the use of CD20 monoclonal antibody rituximab for treating diffuse alveolar hemorrhage in children. This study provides insights into the potential therapeutic applications of rituximab in this rare condition.
AgomAb Therapeutics sets its IPO price at $16 per American depositary share, aiming to raise funds for debt repayment and corporate expenses. The biotech firm focuses on immunology and inflammatory diseases, testing market demand alongside other recent IPOs.
AbbVie is exploring the potential of psychedelic therapies as Johnson & Johnson's esketamine nasal spray, Spravato, continues to see increasing sales. This trend indicates a growing interest in the psychedelic market among pharmaceutical companies.
Biogen is focusing on its late-stage pipeline for multiple sclerosis as it faces increasing generic competition. The company is actively seeking partnerships to enhance its growth strategy.
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