Rare Disease-Focused Rallybio Inks Development Pact With Johnson & Johnson, Stock Shoots Higher
Rallybio Corporation RLYB shares are trading higher after the company announced a collaboration with Johnson & Johnson JNJ to support the development of complementary therapeutic approaches aimed at reducing the risk of fetal and neonatal alloimmune thrombocytopenia (FNAIT). FNAIT is a rare immune…#rallybiocorporation #johnsonjohnsonjnj #fnait #rallybio #johnsonjohnson #johnsonjohnsons #phase2 (Source: Reuters: Health)
Source: Reuters: Health - April 11, 2024 Category: Consumer Health News Source Type: news

Vertex Pharma ’s $4.9 billion takeover of Alpine Immune expected to boost its rare-disease portfolio
Vertex Pharmaceuticals Inc.’s acquisition of Alpine Immune Sciences Inc. makes strategic sense and will bolster the company’s rare-disease franchise, analysts said Thursday. (Source: Reuters: Health)
Source: Reuters: Health - April 11, 2024 Category: Consumer Health News Source Type: news

Woman's rare disease causes 20-pound tumor to grow out of her neck - the mass is so large she carries it around in a sling
WARNING: Story contains graphic images. A 30-year-old woman from Germany with a rare disease that causes growths on the body has a tumor so large it is 20 percent of her body weight. (Source: the Mail online | Health)
Source: the Mail online | Health - April 11, 2024 Category: Consumer Health News Source Type: news

The United Mitochondrial Disease Foundation Announces Inaugural Venture Philanthropy Investment; The Mito Fund Invests $500k in Pierrepont Therapeutics, Inc.
The United Mitochondrial Disease Foundation's venture philanthropy initiative, The Mito Fund has invested $500,000 in Pierrepont Therapeutics, Inc., a company focusing on treatments for primary mitochondrial and rare diseases, for the development of PTI-501, a proprietary intracellular... (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - April 4, 2024 Category: Pharmaceuticals Tags: NPT VEN Source Type: news

First Treatment Found for Rare Disease That Can Lead to Amputation
THURSDAY, April 4, 2024 -- There ' s a glimmer of hope for people afflicted by a rare artery-hardening disease than can lead to amputation. An existing medication called etidronate appears to help slow the buildup of calcium in arteries that ' s a... (Source: Drugs.com - Daily MedNews)
Source: Drugs.com - Daily MedNews - April 4, 2024 Category: General Medicine Source Type: news

Woman's 'one-of-a-kind' disease causes thousands of TUMORS to grow all over her body- including in her nose and mouth
A woman from Trinidad with a rare disease that causes thousands of life-threatening tumors to grow everywhere on her body traveled to a surgeon in LA that could remove the growths. (Source: the Mail online | Health)
Source: the Mail online | Health - April 4, 2024 Category: Consumer Health News Source Type: news

Texas woman, 28, with extremely rare disease that means her hands are permanently swollen reveals struggles of daily life
Lindsey Johnson Edwards, 28, of Texas, was diagnosed with CLOVES syndrome as a child. Only 200 people around the world have been identified with the condition, which caused hand deformities. (Source: the Mail online | Health)
Source: the Mail online | Health - April 2, 2024 Category: Consumer Health News Source Type: news

Groundbreaking gene therapy offers ‘glimmer of hope’ for children with rare diseases
Within the first few weeks after Lori and Lee Greenwood ’s daughter Noa was born, her parents knew that something was wrong. A lot of different things, actually. The infant cried inconsolably. She wouldn’t follow her parents with her eyes, leading to concerns about her vision. She struggled to gain weight and missed milestone after milestone. She was slow to even respond to her parents’ presence. After weeks and months of having doctors dismiss it as the normal fits and fuss of a newborn, the… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - April 1, 2024 Category: Pharmaceuticals Authors: UMass Chan Medical School Source Type: news

PET/MRI may improve diagnosis of Cushing disease
PET/MRI could become the diagnostic method of choice over MRI alone for identifying small pituitary tumors associated with Cushing disease, according to a study published March 21 in the Journal of Nuclear Medicine. In patients diagnosed with the disease yet who had inconclusive MRI results, PET/MRI was positive in 100% of cases, noted lead author Ilanah Pruis, a doctoral student at Erasmus University Medical Center in Rotterdam, Netherlands. “This multimodal imaging technique provides a welcome improvement for diagnosis, planning of surgery, and clinical outcome in patients with Cushing disease,” the authors wrote. ...
Source: AuntMinnie.com Headlines - March 29, 2024 Category: Radiology Authors: Will Morton Tags: Molecular Imaging Source Type: news

Zevra Therapeutics Announces Top-Line Data from the Phase 2 Clinical Trial of KP1077 for Idiopathic Hypersomnia
Top-line data provide key information for the design of a Phase 3 study CELEBRATION, Fla., March 26, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. ZVRA (Zevra, or the Company) a rare disease therapeutics company, today announced top-line data from its placebo-controlled, double-blind Phase 2…#celebration #fla #zevra #phase2 #nct05668754 #kp1077 #zevraphase2 #ih #christopherdrake #phd (Source: Reuters: Health)
Source: Reuters: Health - March 26, 2024 Category: Consumer Health News Source Type: news

Inozyme Pharma to Report Topline Data from Ongoing Phase 1/2 Trials of INZ-701 in Adults with ABCC6 Deficiency and ENPP1 Deficiency on April 8, 2024
Company to host conference call and webcast featuring key opinion leaders on Monday, April 8, 2024 at 8:00 a.m. ET - BOSTON, March 26, 2024 (GLOBE NEWSWIRE) -- INZY ("the Company" or "Inozyme"), a clinical-stage rare disease biopharmaceutical company developing novel therapeutics for the treatment…#boston #inzy #inozyme #easterntime #phase12 #abcc6deficiency #pxe #enpp1 #yjoyceliao #phd (Source: Reuters: Health)
Source: Reuters: Health - March 26, 2024 Category: Consumer Health News Source Type: news

Trial results for Dravet drug spurs stock surge at Bedford biotech
Stoke Therapeutics, which develops RNA-altering drugs for rare diseases, announced what it called “landmark” results from an early-stage trial of a drug for a rare, debilitating form of epilepsy that affects children. (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - March 26, 2024 Category: Pharmaceuticals Authors: Don Seiffert Source Type: news

The World ’s Most Expensive Drug Is Now a $4.25 Million Gene Therapy
A new gene therapy for an ultra-rare disease will have a wholesale cost of $4.25 million, making it the world’s most expensive drug. The one-time treatment, Lenmeldy, won U.S. regulatory approval on Monday to correct the underlying cause of a hereditary condition called early-onset metachromatic leukodystrophy, or MLD. MLD is a fatal disease in which infants sometimes start to lose the ability to walk and talk. Orchard Therapeutics said the drug’s price “reflects its clinical, economic and societal value” in a statement Wednesday.  [time-brightcove not-tgx=”true”] Read Mo...
Source: TIME: Health - March 21, 2024 Category: Consumer Health News Authors: Gerry Smith/Bloomberg Tags: Uncategorized healthscienceclimate wire Source Type: news

Rocket Pharmaceuticals Shines Spotlight On Rare Diseases
Rocket Pharmaceuticals celebrated its sixth annual Rare Disease Day event bringing together leaders in the biotech community along with patients and advocates. (Source: Forbes.com Healthcare News)
Source: Forbes.com Healthcare News - March 19, 2024 Category: Pharmaceuticals Authors: Yola Robert, Senior Contributor Tags: ForbesWomen /forbeswomen Leadership /leadership Innovation /innovation Healthcare /healthcare standard Source Type: news

Pittsburgh rare disease company tops patient satisfaction survey
RareMed Solutions tops patient satisfaction survey (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - March 18, 2024 Category: Pharmaceuticals Authors: Ethan Lott Source Type: news